Pembroke mom is on a mission to give hope

Posted: Published on July 22nd, 2013

This post was added by Dr Simmons

New England Patriots players surrounded her, yet Christine McSherry of Pembroke could not take her eyes off 10-year-old Max as he ran around like a normal kid at a Patriots training camp event last August.

Max, like McSherrys own 17-year-old son, Jarrett, suffers from Duchenne muscular dystrophy a disorder, primarily seen in boys, that is caused by a mutation in the gene that codes for the muscle protein dystrophin.

Other forms of muscular dystrophy cause a decrease in dystrophin, but boys with Duchenne do not produce any of the protein. Many of them are confined to a wheelchair by age 12.

There is no approved treatment for Duchenne muscular dystrophy, which ultimately causes early death.

But as McSherry watched the hopping and skipping of Max, a boy whose strength was quickly decreasing a year prior, she felt reason for hope.

There was a significant difference, McSherry said Friday. This kid, you would have no idea there was anything wrong with him.

The hope comes in the form of eteplirsen, a clinical drug made by Cambridge-based Sarepta Therapeutics. It has shown great potential for stopping the advance of Duchenne.

Founder of the Jett Foundation for fighting Duchenne muscular dystrophy, McSherry is taking to Capitol Hill to advocate for the accelerated approval of eteplirsen by the U.S. Food and Drug Administration.

A letter in support of the accelerated application was sent Friday to the FDA. It bore the signatures of 32 members of Congress.

On July 9, McSherry spoke at a congressional briefing hosted by U.S. Rep. William Keating. Sen. Elizabeth Warren is collecting signatures from U.S. senators to send to the FDA before it meets this week with Sarepta Therapeutics to discuss pursuing an accelerated application.

See the article here:
Pembroke mom is on a mission to give hope

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