Rare Disease

08 Aug 2012
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Drug Approved For Saskatchewan Girl With Rare Disease

Sticky Post By On 8 August, 2012

Health Care-PB-Regina-Violet Revet, sick child-Aug 2 2012-1Naglazyme will extend the little girl’s life, was not previously available in the province
Reported by News Talk Radio staff
First Posted: Aug 8, 2012 7:25pm
Change text size: + –

Violet Revet, a three-year-old girl from Langenburg, is one of only nine people in Canada who has Mucopolysaccharidosis VI (MPS-VI) and now she will receive treatment she desperately needs.

MSP-VI has no cure and the longer she goes without treatment, the shorter she could live.

Andrew McFayden’s son, Isaac, was diagnosed with the disease in 2005.

“It’s a very devastating condition. Essentially kids that are diagnosed with MPS lack an enzyme in their blood that breaks down all the complex sugars. Because those complex sugars that occur naturally in the body don’t break down they need to find a place to go and so they end up being stored in the bones, tissues, organs, and muscles and it leads to many progressive symptoms: heart and airway disease, progressive stiffening of the joints, corneal clouding, spinal cord compression.”

“Essentially every muscle, tissue, and organ in the body is affected by this disease with the exception of the brain.”

McFayden was told Isaac would only have seven or eight years to live. That was before doctors realized a treatment was making the rounds in the UK and the US, a synthetic enzyme known as naglazyme that acts as a replacement in the body.

Isaac could get access to it under a special access program but the drug wasn’t covered in Ontario. It took McFayden eight months of lobbying before the provincial government agreed to pay for it.

Violet’s family asked Saskatchewan’s health ministry for funding so she could get the same treatments.

Today, the government drug review process has approved naglazyme and her doctor will begin administering the treatment.

With files from News Talk Radio’s Patrick Book

08 Aug 2012
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Violet Revet, Saskatchewan Girl With Rare Disease, Will Have Her Treatment Paid For By Province

Sticky Post By On 8 August, 2012

Violet RevetCP  |  By  Posted: 08/08/2012 9:40 pm Updated: 08/08/2012 10:23 pm

Violet Revet of Langenburg is one of only nine people in Canada who has Mucopolysaccharidosis VI. ALAMY

REGINA – The Saskatchewan government has agreed to pay for treatment for a three-year-old girl with a rare disease.

Violet Revet of Langenburg is one of only nine people in Canada who has Mucopolysaccharidosis VI.

The disease has no cure and the longer she goes without treatment, the shorter her life span will be.

Andrew McFayden’s son, Isaac, was diagnosed with the disease in 2005.

McFayden, of Campbellford, Ont., was told Isaac would only have seven or eight years to live.

That was before doctors realized a treatment was making the rounds in the United Kingdom and the United States — a synthetic enzyme known as naglazyme that acts as a replacement in the body.

Isaac could get access to it under a special access program but the drug wasn’t covered in Ontario.

It took McFayden eight months of lobbying before the provincial government agreed to pay for it at a cost of about half a million dollars a year.

“It’s a very devastating condition,” said McFadyen, whose eight-year-old son goes to Toronto for regular treatments.

“Essentially, kids that are diagnosed with (it) lack an enzyme in their blood that breaks down all the complex sugars. Because those complex sugars that occur naturally in the body don’t break down they need to find a place to go and so they end up being stored in the bones, tissues, organs, and muscles and it leads to many progressive symptoms: heart and airway disease, progressive stiffening of the joints, corneal clouding, spinal cord compression.”

McFadyen has said that while the treatment is not a cure, it will keep the children alive while efforts to find a cure are made.

Violet’s case had prompted many supporters to flood Premier Brad Wall’s Twitter account with pleas for the funding, and Wall asked the ministry to look into the matter.

On Wednesday the approval came through.

(CJME)

07 Aug 2012
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Every Moment is a Gift for the McFadyen Family

Sticky Post By On 7 August, 2012

The following is part one of an interview with Andrew McFadyen, the father of eight-year-old Isaac who has the rare disease MPS VI. Part two will feature the McFadyen family’s advocacy for other children diagnosed with the disease. The third part will announce their lofty new fundraising project.

CAMPBELLFORD – A year ago this time, Andrew McFadyen would have said his oldest son, Isaac, was the healthiest he had ever been.

Even in October, the McFadyen family from Campbellford was in part celebrating that success at The Isaac Foundation’s second annual Gala For A Cure in Cobourg.

“Three weeks after that we got word that his heart wasn’t doing all that well,” Andrew McFadyen said during an interview earlier this week. “Then in early March, he had some sort of brain event that we don’t really know what it was. There were indications that it could have been a stroke. So we’ve been spending a lot more time than normal at the hospital. We spend a lot of time there anyway but we were doubling and tripling up on our trips and having a lot of emergency scans and emergency tests.”

Isaac McFadyen, who is now eight years old, has lived with MPS VI (Maroteaux Lamy Syndrome) since 2005. He is one of only nine confirmed cases in Canada — and about 1,100 worldwide — of the disease which is caused by a rare enzyme deficiency. Andrew McFadyen and his wife, Ellen, established The Isaac Foundation in 2006 to help raise awareness of the disease and money to help fund innovative research projects.

Having watched his oldest child live with the progressive disease since 2005, McFadyen said every day is a blessing for himself and Ellen, who also have a younger son, Gabriel.

“When we were told he might not live past the age of seven, every moment with him is a gift and we recognize that and take it all in and it’s really special for us,” he said.

This story about the McFadyen family isn’t new, but rather more of a reintroduction.

For those who don’t know, symptoms of MPS VI include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span.

“Children who have this condition lack an enzyme in their blood that breaks down all of the complex sugars,” McFadyen explained. “We have the complex sugars that build up naturally in our body, it has nothing to do with the foods that we’re eating or the diet that we’re on. They just build up naturally in our body and are broken down by all the enzymes in our blood. Children that suffer from MPS lack one of the enzymes responsible for breaking down those complex sugars, so instead of breaking down, they find a place to go and store. They store up in the bones, the tissues, the organs, the muscles…very progressively throughout the course of the affected individual’s lifespan.”

As devastating as the news of the diagnosis was all of those years ago, there was still a rollercoaster of emotions to follow.

First, there was hope when they found out a treatment was available to slow the progression of the symptoms, but at a price tag of a million dollars a year that the Ontario government would have to agree to pay for.

“Parents have to endure the devastating news that their child has been diagnosed with this condition. I was sick for weeks and lost 40 pounds. It’s not news I would wish on any parent ever,” McFadyen said. “We were told at the time that Isaac wouldn’t make it past his early teens and that even by seven years old he might be gone from us. That was really, really hard for both my wife and I to take in and handle.

“Then this idea that we found there’s a treatment that can stave off the disease, slow it down…that’s incredibly hopeful and you want to do everything in your power to ensure your child can get this treatment. But then there’s this hook that yes you can bring it to Canada so long as the Ontario government says they’ll pick up the price tag of a million dollars.”

While not a cure for MPS VI, the life-sustaining treatment available is called Naglazyme, which is an Enzyme Replacement Therapy designed to provide patients with a synthetic version of the enzyme they are lacking. Small doses are infused into the patient’s bloodstream.

Naglazyme is only available to Canadian patients through the federal government’s Special Access Program.

Initially, the McFadyens request to the provincial government was denied.

“Being told no from your government, no we’re not going to pay to save your child’s life is devastating, it’s heartbreaking, it’s worrisome…it makes you question the priorities of the people in charge.

“That was really hard, in one sense trying to cope and trying to just come to terms with your new circumstance in life and then to turn around and not have your government look after you was disappointing and discouraging for us.”

They didn’t give up, though, and continued to lobby the government. With the help of former Conservative MPP Elizabeth Witmer, who was the opposition’s health critic at the time, they were ultimately successful (more on the initial rejection and subsequent approval in part two of this interview tomorrow).

Isaac heads to Sick Kids in Toronto a weekly basis and the treatment has altered his life.

“Had he not been on this treatment, he would be suffering from a lot of very, very devastating symptoms,” McFadyen said.

Despite the setbacks during the past year, McFadyen said he believes, and hopes, that Isaac’s condition is stable again.

“We always have hope that’s the case, but we know that we have to work really hard and find a cure and other treatments because as we’ve known from the start this treatment isn’t a cure,” he remarked. “You can only stave off the disease, or slow it down for so long. There’s always going to be this disease building up in his body and so I guess this past year has really just re-motivated us and re-energized us to ensure that we can find a cure for him and everyone who has this disease.”

McFadyen said there’s a “tricky balance” of developing a sense of normalcy when things are going well.

“It just becomes an everyday part of your life — our weekly trips to Toronto, his receiving this very expensive medication that seems to be doing really well — and then you’re blind-sided with what you think is a routine heart appointment. You’re blind-sided when his schools calls and says he’s suffering this incredible headache and can’t see out of his eye,” he said. “That blind-sided news takes you right back to the start again. That’s what we’ve been going through this past year. We keep telling ourselves we’ll never let our guard down again, but sometimes it’s okay that we do because that means things are going really well and he’s doing okay.”

Isaac’s parents want him to live as normal a life as he possibly can.

“We don’t want Isaac ever to just be identified as the sick little boy,” McFadyen said. “We want Isaac to be identified for who he is and what he does and the fact that he just happens to have this rare disease.”

jeff.gard@sunmedia.ca

twitter.com/NT_jgard

 

02 Aug 2012
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Premier Agrees To Look Into Treatment For Girl

Sticky Post By On 2 August, 2012

Joe Couture, The StarPhoenix

Published: Thursday, August 02, 2012

After a Twitter campaign urged him to get involved, Premier Brad Wall said on the social networking site the Health Ministry has been asked to look into the case of a three-year-old Saskatchewan girl dealing with a rare disease.

A search for @PremierBradWall Wednesday resulted in a feed full of tweets from the last several days directed at the premier – who was travelling with his family back to Saskatchewan – asking him to ensure Violet Revet, a Langenburg girl, receives treatment for a rare enzyme deficiency called MPS VI, or Maroteaux-Lamy syndrome.

“The minister’s office has been asked to look into it,” Wall tweeted in reply to Ontario-based Andrew McFadyen of the Isaac Foundation, who organized the campaign and put out a news release later in the day. McFadyen’s son, Isaac, has the same condition as Revet and the foundation raises money for research and advocates for families of those with the disease.

“Public pressure on governments, we know, works,” McFadyen said in an interview Wednesday about the Twitter effort, noting many people are interested in the Isaac Foundation and have been involved in campaigns to ensure those with the disease receive treatment.

McFadyen said sufferers of MPS VI lack an enzyme in their blood that breaks down cellular waste, which instead builds up in bones, tissues, organs and muscles, leading to many symptoms including heart and airway disease, corneal clouding, stiffening of joints, shortened stature and premature death. There are nine confirmed cases in Canada and about 1,100 worldwide.

Though there is no known cure for MPS VI, McFadyen said a therapy exists, designed to provide a synthetic version of the missing enzyme through weekly doses. The cost can range from $300,000 per year for a child to $1 million per year for a young adult.

The treatment is only available to Canadian patients through a federal special access program, but is being used for patients in Ontario, British Columbia and Quebec through that program and is funded by the provincial governments, McFadyen said.

“Isaac was the first person in Canada to successfully bring this treatment here and it’s done wonders,” he said. “It’s not a cure. It’s more of a lifeboat.”

Tracey Smith, director of pharmaceutical services with the Saskatchewan Drug Plan, said Wednesday the request is being considered.

“We hope to have a decision in the next couple of weeks,” Smith said.

“We are aware of this request and the request for coverage of this particular drug was made to the drug plan on July 9 of 2012,” Smith added. “We do have a mechanism for reviewing these types of requests and we’re basically looking at the request and are working as quickly as we can to make a decision.”

A variety of factors are examined when considering a request like this one, she said, noting those include effectiveness of the drug, treatment guidelines, safety information and the cost impact. McFadyen said it took 18 months for Violet to be diagnosed and seven months from her diagnosis for the application to be received by the ministry. He urged quick action, noting long appeal processes have been required in other provinces where the treatment has eventually been approved.

“There is zero guarantee that this decision is going to be positive for Violet and her family,” he said.

The Revet family was travelling to Saskatchewan from Ontario on Wednesday and could not be reached.

jcouture@thestarphoenix.com

02 Aug 2012
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Saskatchewan Girl Waiting For Help With Rare Disease

Sticky Post By On 2 August, 2012

Health Care-PB-Regina-Violet Revet, sick child-Aug 2 2012-1Her family is asking for help with the treatment from the provincial government

Reported by Patrick Book
First Posted: Aug 2, 2012 3:26pm | Last Updated: Aug 2, 2012 3:45pm
Change text size: + –

Violet Revet is a very sick little girl and her family is worried the province isn’t moving quickly enough to help her.

The three year old from Langenburg is one of only nine people in Canada diagnosed with

Mucopolysaccharidosis VI (MPS-VI). The ultra-rare disease has no cure and the longer Revet goes without treatment the shorter her life could be.

“Violet right now without treatment is definitely going to suffer and that’s really devastating for the family,” explained Andrew McFayden in an interview Wednesday from his home in Ontario.

McFayden knows all too well the impact of MPS-VI. His son Isaac was diagnosed with it in 2005. McFayden said the disease ravages the body if left untreated.

“It’s a very devastating condition. Essentially kids that are diagnosed with MPS lack an enzyme in their blood that breaks down all the complex sugars. Because those complex sugars that occur naturally in the body don’t break down they need to find a place to go and so they end up being stored in the bones, tissues, organs, and muscles and it leads to many progressive symptoms: heart and airway disease, progressive stiffening of the joints, corneal clouding, spinal cord compression. Essentially every muscle, tissue, and organ in the body is affected by this disease with the exception of the brain.”

McFayden was told Isaac would only have seven or eight years to live. That was before doctors realized a treatment was making the rounds in the UK and the US, a synthetic enzyme known as naglazyme that acts as a replacement in the body.

Isaac could get access to it under a special access program but the drug wasn’t covered in Ontario. It took McFayden eight months of lobbying before the provincial government there agreed to pay for it and he suspected the delay in Violet’s case is the same as the delay there.

“It’s (up to) a million dollars a year, so I understand that governments have to look closely at this type of thing. Usually what they say they have to look at is the safety of it but it’s already available in the United States, most of the UK…many, many other countries have this. The safety has been proven. Essentially it comes down to dollars and cents and (I believe) that’s why these provinces are delaying.”

Thanks to weekly treatments many of isaac’s symptoms were reversed. It’s given the boy a new lease on life as his family hopes that a cure could one day be found.

Asking for help

After her diagnosis Violet’s family made a request to Saskatchewan’s health ministry for funding so she can get the same treatments.  After going more than six months without an answer they went to McFayden for help and he says time is very much of the essence.

“Early detection and early treatment of this conditions is paramount to the future success of children with MPS-VI,” he asserted.

In an effort to spur the government to action McFadyen and supporters of The Isaac Foundation, an advocacy and fund raising group he started to help others with the disease, have been lobbying Premier Brad Wall via the social networking web site Twitter.

On Wednesday the Premier responded, saying they had his attention and revealing that he had directed the Health Ministry to contact the Revet family director. But McFadyen says lip service doesn’t do a sick little girl any favours.

“In Canada we’re supposed to have equal and fair access to health care and right now Violet is not receiving that.”

Tracey Smith is the director of pharmaceutical services for the ministry. She said the delay it is not about the cost of the drug. She denied that any unnecessary delays have happened, insisting the government didn’t get the family’s funding request until July 9th.

“We received the request that day and we have been reviewing the request. We’re working as quickly as possible in order to make a decision and communicate that back to the family.”

She said every funding application goes through a similar review process but she stressed the ministry is looking at the fact that the treatment is already paid for in Ontario, Quebec, and B-C, among other things.

Smith said she’s hopeful a decision will be made in a few weeks but she won’t say what the likelihood of approval is.

McFayden is sorry to hear it; he said work needs to begin as soon as possible because pharmacists and hospital staff that will be working with Revet will need weeks of training before treatments can begin.

Edited by CJME’s Lisa Schick.

01 May 2012
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School Supports Student With Rare Condition

Sticky Post By On 1 May, 2012

1337885928545_ORIGINALMichelle McCarthy for QMI Agency

CAMPBELLFORD – Students at Kent Public School energetically poured into the play yard recently for a Jump-A-Thon fundraiser in Support of the Isaac Foundation.

The event which included hula hoop contests, ball hockey and music, was just one of many fundraising initiatives that have been held for Kent’s own Grade 2 student Isaac McFayden. While energetic and full of life, Isaac lives with MPS VI, a disease so rare it affects only five children throughout Canada.

MPS VI is a progressive disease that’s caused by an enzyme deficiency. It creates a variety of symptoms that can affect every aspect of the body from growth and internal organs, to muscles and joints. There are various degrees and types of MPS, such as the MPS VI that afflicts young Isaac.

“Our world was definitely changed forever in 2005 when at 18 months Isaac was diagnosed with this devastating disease. It was very hard on both me and Lisa, who at the time was pregnant with Gabriel, our second child,” said Andrew McFayden, director of the foundation and Isaac’s dad.

Although Isaac’s brother Gabriel was not born with MPS, at the time the McFaydens were faced with a very real one-in-four chance that their new baby would also be born with the genetic disease.

“It was a hard time and we both took time off work but we spent some time feeling sorry for ourselves and for Isaac, we realized he wasn’t going to get better with us sitting around watching him deteriorate, which is what began the Isaac Foundation.”

The McFaydens have been working tirelessly ever since to help their son and others afflicted with this rare condition. Andrew McFayden said that, luckily for Isaac’s sake, the foundation was able to achieve a lot in a small amount of time. In 2005 when this began, the lifesaving treatment available to slow the progression of MPS was only available in the US and was far too expensive to afford, costing millions of dollars a year to administer.

“Our first goal was to get the treatment brought to Canada and then to have the province fund it fully,” McFayden said. “It was a long and hard fight but we were successful and it has made all the difference in all our lives and continues to help maximize Isaac’s quality of life.”

Since the Isaac Foundation’s inception in 2006, it has donated more than $300,000 to research and is close to committing another $60,000 towards finding a cure for Isaac and others who are suffering from this rare condition. McFayden said it’s thanks to continual support, like that at Kent Public School, that they are able to continue achieving their goals at the foundation.

Kent Public School’s May 17 Jump-A-Thon raised more than $1,000 for the foundation, which is a nice addition the $12,000 the school has already raised over the last three years through other endeavours such as this.

1337885928721_ORIGINALThe school secretary, known to most as Ms. Lisa, spoke very highly of the young student and the bond and sense of community he seems to help foster at the Campbellford public school.

“Although Isaac’s illness causes him to face certain challenges, he never comes across or presents himself as if he has any challenges,” said Ms. Lisa. “He’s just so personable and smart, and has such a great attitude and joy for life that it’s really inspirational to watch.”

She said as a school, Kent is often asked to support a number of charities all very worthy of support, but it’s particularly great when they get an opportunity to help out with something so close to home.

“Isaac and our fundraisers have brought us so much closer as a school and have fostered such a sense of community among the students and staff,” said Ms. Lisa. “All you have to do is look out in the yard and see them interacting with Isaac, watch the participation, excitement and acceptance and it’s like that every time we hold an event for the Isaac Foundation.”

McFayden said the Isaac Foundation has a few of their bigger fundraisers coming up over the next few months including their main Gala for the Cure on Oct. 13, 2012 and their charity golf tournament on June 23.

He added this coming May 26 and 27 the Isaac Foundation will be participating in Ottawa’s annual race weekend in an effort to raise funds for the foundation. A local running team of more than 35 individuals have been fundraising and collecting sponsors leading up to the event, and McFayden said it will be interesting to see how much money the foundation can raise.

“The research we help fund is beyond important and looks very exciting. Some of it quite promising for Isaac and others in the shorter term so we’re in this to really do everything we can to continue funding the MPS research taking place across the globe,” he said.

1337885928633_ORIGINALFor more information on the Isaac Foundation, Isaac and upcoming fundraising events, visit www.theisaacfoundation.com.

11 Feb 2012
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Concert at Campbellford Has Dual Purpose

Sticky Post By On 11 February, 2012
Feb 11, 2012

Raise money for charity, support local food

Queen concert

Submitted photo

CAMPBELLFORD — Singer/songwriter Andrew Queen will give a Family Day concert at the Aron Theatre to promote his new CD that touts the virtues of buying food locally. It’s also a fundraiser for Isaac McFayden, between Mr. Queen and his two sons, Lewis and Ivan. Isaac’s parents established a foundation to raise money for research to treat and cure the rare disease their son has, MPS VI. January 2012
Northumberland NewsTRENT HILLS — Andrew Queen is a man on a mission, two of them in fact, and they’ll come together Feb. 20 at the Aron Theatre when the popular children’s entertainer performs songs from his newest release, “GROW.”

One goal he’s set for himself is to get kids singing songs about eating healthy food grown locally, the other is to raise money for a Trent Hills family’s son who has a rare, progressive disease, MPS VI, caused by an enzyme deficiency.

All proceeds from the sale of tickets to the Family Day concert and a portion of the CD sales will be donated to the Isaac Foundation that Ellen and Andrew McFayden set up after their son Isaac, now seven, was diagnosed with MPS VI in October 2005. The registered charity supports research into innovative treatments and potential cures for Maroteaux-Lamy syndrome, which can stunt growth, impair vision, cause stiffness in joints, affect breathing, and enlarge the liver or spleen.

“GROW is an album about community-building and acting local,” Mr. Queen said in a press release. “The McFaydens have demonstrated such dedication, grace, love and strength in the face of unbelievable challenges. I can’t think of a better way to celebrate Family Day and my album release than recognizing this family and supporting their research efforts.”

Mr. Queen raised money for local causes at two previous Family Day concerts; the first was for the music program at Kent Public School, the second for improvements at Kennedy Park, he told The Independent.

His wife, Karen Stille, suggested The Isaac Foundation this time out. The McFaydens were one of the first families the couple met when they moved to Trent Hills seven years ago, and a benefit they attended last “was really a moving event,” he said.

Ms. Stille also wrote most of the lyrics for the songs on the new CD.

“We’ve turned into a songwriting dynamo,” said Mr. Queen, whose second CD, “Too Tall,” won the 2010 Canadian Folk Music Award for Children’s Album of the Year and received a Parents’ Choice Recommended Award. One of its songs, “Big Troubles for Little Red,” placed second in the 2009 International Songwriting Competition.

“GROW” promotes buying local and supporting local family farms, Mr. Queen said. Its point of view is captured in a new music video that will be premiered at the concert. “Just Down the Road” was filmed and produced by Stephen Rapos of Fiddlehead Studios in Warkworth.

“The big message is to get kids thinking about where their food comes from and thinking about eating healthy,” Mr. Queen said. All kinds of fruits, vegetables, meats, cheeses, breads, butter, and preserves are available “within a short drive,” and the area people responsible for their production deserve to be supported.

“It’s a good feeling,” he said. “You can start to feel a little control over the food that’s coming onto the table.”

The concert begins at 2 p.m. at the Aron. Mr. Queen will be joined on stage by local musicians Tim Hadley (upright bass), Luke Mercier (fiddle/banjo), Janet Mercier (vocals) and Ken Layton (drums).

Tickets can be purchased for $5 at the Aron Theatre, Kerr’s Corner Books, The Grindhouse Café and the Campbellford-Seymour Community Foundation, or for $7 at the door. To learn more about Mr. Queen and The Isaac Foundation, visit www.andrewqueen.ca and www.theisaacfoundation.com.

09 Feb 2012
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Family, Music, and Charity

Sticky Post By On 9 February, 2012

-253864_ORIGINALCommunity Press staff

Campbellford – If music is food for the soul, then Andrew Queen’s latest release, GROW, plays a dual role.

The CD offers up what he calls “generous helpings of original tantalizing tunes, seasoned with sweet sing-a-longs and sprinkled with silliness,” while getting kids to sing about growing, sharing and eating local, healthy food.

“GROW is an album about community-building and acting local,” said Queen, who will perform songs from his new CD during a Family Day Performance at the Aron Theatre Cooperative.

Queen said he has chosen the Aron Theatre Cooperative as the venue for the release of GROW because of the “vibrant community involvement that keeps the theatre thriving.”

All proceeds from ticket sales and a portion of CD sales will go to a local registered charity, The Isaac Foundation, which was created by Ellen and Andrew McFayden after their son, Isaac, was diagnosed with MPS VI, a rare, progressive disease caused by an enzyme deficiency. Their mission is to raise funds to support research into innovative treatments and potential cures for MPS VI and other MPS diseases, to increase public awareness about MPS diseases, and to provide support and information to other families dealing with this devastating diagnosis.

“The McFaydens have demonstrated such dedication, grace, love and strength in the face of unbelievable challenges,” Queen said. “I can’t think of a better way to celebrate Family Day and my album release than recognizing this family and supporting their research efforts.”

Beginning at 2 p.m. Feb. 20, Queen will take the stage with local musicians Tim Hadley, playing upright bass, Luke Mercier, playing fiddle and banjo, vocalist Janet Mercier and Ken Layton on drums. During the live performance Queen will screen a new music video for his family farm anthem, “Just Down the Road,” filmed and produced by Warkworth resident Stephen Rapos, of Fiddlehead Studios.

Advanced tickets – at $5 per person – are recommended. Tickets at the door are $7. Tickets are available at the Aron Theatre, Kerr’s Corner Books, The Grindhouse Cafe, and the Campbellford-Seymour Community Foundation. For more information, visit www.andrewqueen.ca and www.theisaacfoundation.com.

30 Sep 2011
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Sarah Harmer To Perform For Charity Gala In Cobourg

Sticky Post By On 30 September, 2011
Sep 30, 2011

Sarah Harmer/Isaac Foundation Gala

Submitted photo

COBOURG — Sarah Harmer will be performing at the 2nd annual Isaac Foundation Gala being held Saturday, Oct. 1 at Cobourg’s Best Western Ballroom. The Isaac Foundation is a not-for-profit organization that funds research aimed at finding a cure for children’s MPS VI.
Northumberland NewsCOBOURG — Juno award-winning musician Sarah Harmer will be lending her voice to a local fundraising gala for the Isaac Foundation.

Formed in 2006 by Andrew McFayden and Ellen Buck-McFayden after their son, Isaac, was diagnosed with mucopolysaccharidosis VI (MPS VI), the foundation funds innovative research projects that aim to find a cure for children’s MPS VI.

MPS VI is caused by the lack of an enzyme in the blood that breaks down complex sugars which then build up in the tissue, organs, and bones and leads to, among other things, hydrocephalus, heart and airway disease, spinal cord compression, stiffening joints and stunted growth.

Isaac was diagnosed with the disease when he was 18 months old. At the time, he was only the third child in Canada diagnosed with MPS VI and there was no approved treatment. Luckily, approval came shortly after Isaac’s diagnosis and he began treatment that he continues to this day.

“It’s not a cure but it’s his life-boat until a cure is found,” said Mr. McFayden. “And Isaac has that future back that he never had without treatment.”

Since the foundation formed, it has raised $240,000 for research and by the end of the year Mr. McFayden plans to make it $300,000. With most of their major fundraising events happening outside their home-base area, Mr. McFayden said they had always wanted to have a gala and thought it was the perfect event to have close to home.

“This is the second year we’re having the gala,” he said. “Last year was a great success, Ron Sexsmith performed and we were able to raise $20,000. This year we’re hoping to raise $40,000.”

A great deal of international support and recognition has come from big names such as musician John Mayer and Major League Baseball star Roy Halladay, who both have become close friends of Isaac and the foundation.

John Mayer, who Mr. McFayden said often lends a hand to raise awareness of the organization during his concerts and on his website, will be the recipient of this year’s Foundation ‘Lifetime Impact Award’, an award presented to a person who has made a positive impact on and increased Isaac’s quality of life as he fights MPS VI.

Last year’s recipient was Kitchener-Waterloo MPP Elizabeth Witmer, who tirelessly advocated to secure funding from the Ontario government to fund the life-sustaining treatment that Isaac receives at The Hospital For Sick Children on a weekly basis, said Mr. McFayden.

“We’re always so humbled with people and their desire to help,” said Mr. McFayden. “People seem to read about Isaac’s story and they want to help, we’re really very fortunate and couldn’t ask for more.”

The Isaac Foundation’s 2nd Annual ‘Gala For A Cure’ will be held Saturday, Oct. 1, at the Best Western Ballroom in Cobourg. Tickets for the event, which include a wine tasting by Huff Estates Winery, hors d’oeuvres, dinner, Ms. Harmer’s performance, a silent auction, and dancing, are on sale now and can be purchased by contacting Ellen Buck-McFadyen at 705-395-8854 or ellen@theisaacfoundation.com.

For more information about The Isaac Foundation, Isaac McFadyen, or MPS VI, visithttps://www.theisaacfoundation.com.

13 Sep 2011
Sticky Post By Posted in Media, Newspaper Permalink

Isaac Foundation To Hold Second Gala For A Cure

Sticky Post By On 13 September, 2011

NORTHUMBERLAND – Government funding for live-sustaining treatment for MPS VI (Maroteaux Lamy Syndrome) is already extremely important. The only thing better would be finding a cure for the rare enzyme deficiency disease.

That was only reinforced to the McFadyen family of Campbellford last month when they helped another Ontario family lobby the provincial government for the same funding their own son receives to help battle MPS VI.

Seven-year-old Isaac McFadyen — the son of Andrew and Ellen and brother of Gabriel — is one of eight confirmed cases in Canada (and 1,100 worldwide) who have the progressive disease. Symptoms include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span. The McFadyens established The Isaac Foundation in 2006.

There is no known cure yet for MPS VI, but there is the life-sustaining treatment available called Naglazyme, which is an Enzyme Replacement Therapy designed to provide patients with a synthetic version of the enzyme they are lacking. Small doses are infused into the patient’s bloodstream.

Isaac has been receiving the treatment once a week for five years at the Hospital for Sick Children in Toronto. The cost of the treatment, produced by Biomarin, can range from $300,000 for a small individual to $1 million for a young adult each year. The McFadyens lobbied the provincial government to get funding for the treatment and were ultimately successful.

In August, they helped a Palmerston family do the same. Darren and Pam More’s two-year-old son, Jasper, was diagnosed with MPS VI six weeks ago. Within days of being notified they were denied funding for the treatment, the provincial government reversed its decision.

Both families are grateful for the funding as they aim to find a cure.

The Mores will be attending the second annual Gala For A Cure, presented by The Isaac Foundation, Saturday, Oct. 1 at the Best Western in Cobourg.

Tickets are $100 (receive a $50 tax receipt) and will feature a special intimate musical performance by Juno Award winner Sarah Harmer, as well as wine tasting, dinner and a silent auction.

“Knowing that we desperately need to find a cure for these children makes these events that much more important and that much more special for everyone,” Andrew McFadyen said. “Knowing that every dollar goes directly into research is very important as well.

It’s also important to raise awareness of the disease.

“The last five years of us raising awareness was very crucial in helping Jasper get treatment,” McFadyen said.

Last year’s inaugural gala raised $20,000 and went towards research on behalf of The Isaac Foundation. The success was from those who bought tickets to the event as well as donated items to the silent auction.

“For me it was the community feeling that was there,” McFadyen said. “This sense of caring and wanting to help out. With it being such an intimate evening, I felt it was really special for everyone.”

Singer/songwriter Ron Sexsmith helped make last year’s gala a special evening, McFadyen noted, and he expects Harmer will help create a similar atmosphere. The McFadyens are thrilled to have her be a part of the event. They are friends with The Tragically Hip, who are friends with Harmer, and they also received help from Sexsmith’s wife in bring Harmer to this year’s gala.

“There’s a lot of connections that Sarah and we already had, we just needed to connect the dots in order to make it work,” McFadyen said. “When she looked at the website and learned about The Isaac Foundation, she was more than happy to come and pitch in and be a part of this. We were really touched when we got the call saying yes she’s going to be there and perform, and she’s going to spend the whole night with us.”

Anyone wishing to purchase tickets, or donate items for the silent auction, can contact Andrew by phone at 613-328- 9136 and e-mail at mcfadyena@me.com or Ellen at 705-632-0983 and ellen@theisaacfoundation.com.

Tickets are limited.

jgard@northumberlandtoday.com