Health: New Hope For Duchenne Patients

By Stephanie Stahl

PHILADELPHIA (CBS) -- New hope for families battling a devastating illness.  After months of debate, a much needed treatment is back on the fast track for approval.  3 On Your Side Health Reporter Stephanie Stahl is here with the latest.

It's called Duchenne, a severe form of Muscular Dystrophy, that causes weakness and  difficulty moving.  Until now treatments have been limited.  This week the maker of an experimental drug says it's back on track to get quick approval.

12-year-old Yuva Gambhir depends on a scooter to get around.  The Center City middle schooler is battling Duchenne Muscular Dystrophy.

"It limits my ability to do things that most normal people can do like it's harder for me to walk.  It's harder for me to run," said Yuva.

Duchenne is a rare genetic disorder that causes progressive muscle degeneration and weakness.  It affects mainly boys, and there is no cure.

"This disease is going to get to him.  It is going to wear his body out.  It affects every muscle in your body, that includes your respiratory organs, your heart, everything.  We are just hoping against hope that there is a treatment in time to help him," said Sonal Gambhir, Yuva's mother.

They say their best hope currently lies with an experimental drug called eteplirsen made by Sarepta.

The drug is important because it helps to mitigate the problem in a substantive way," said Manu Gambhir, Yuva's father.

But the FDA, felt there wasn't enough evidence to prove the drug worked well enough and the approval process was stalled, crushing for many families struggling with Duchenne.

They petitioned the FDA pushing for the drug to get fast tracked.

This week the drug company announced a new plan and is working with the FDA to quickly get the drug approved.

"It's very good news.  It's very promising," said Sonal.

"I think it's a pretty cool, and it's going to be a good drug," said Yuva.

If approved, the drug could potentially be available by next summer.  As part of the approval process the company is starting new trials to test the drug, along with other similar medications.

Yuva's family is hosting a fundraiser in May to raise awareness and money for Duchenne research.

Duchenne Muscular Dystrophy Information- http://mda.org/disease/duchenne-muscular-dystrophy/overview

The Race To Yes- http://theracetoyes.org/

Sarepta Clinical Trial Information- http://www.skipahead.com/

Blingo to Cure Duchenne- www.blingophilly.org