Palmer Says New Drug will Help Duchenne Muscular Dystrophy Patients

September 22, 2016

By Brandon Moseley
Alabama Political Reporter

US Representative Gary Palmer (R-Hoover) announced that the US Food and Drug Administration has approved a Duchenne Muscular Dystrophy (DMD) treatment that parents of Duchenne’s patients have been asking for, for a while now.

Congressman Palmer said in a statement, “On Monday, the Food and Drug Administration (FDA) approved a drug for Duchenne muscular dystrophy more than 100 days after the agency’s legally mandated decision date. The drug, Exondys 51 (eteplirsen), is the first of its kind and will benefit patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping.”

Rep. Palmer continued, “This was a victory for those affected by Duchenne muscular dystrophy and their families. With the FDA approval of Exondys 51, some people impacted by Duchenne have new hope for an improved quality of life and a better tomorrow. I applaud all those who have advocated for this medical advancement, including one of my constituents, Gabe Griffin (Hope for Gabe, Inc.). While this medical advancement does not treat the cause of Gabe’s muscle degeneration, it is a step in the right direction towards creating and approving the drug that will.”

Gabe Griffin is an 11-year-old Shelby County resident who was diagnosed with DMD eight years ago.

DMD is a genetic medical condition caused by an absence of dystrophin in the body. This fatal disorder transpires from early childhood and causes progressive muscle weakness that eventually leads to serious medical problems. There is currently no cure for Duchenne. Because the Duchenne gene is found on the X-chromosome, it primarily affects boys (Girls have two X chromosomes so the non-carrier X will normally override the carrier X and prevent expression of the condition). Most DMD patients are confined to a wheel chair by age 12 and most are dead by age 20.

In June 2015, Alabama Governor Robert Bentley (R) signed Senate Bill 357, “Gabe’s Right to Try”, into law. This bill allows patients with a terminal illness the right to authorize access to and use of experimental treatments after they have met certain requirements. SB357 was sponsored by Sen. Cam Ward (R-Alabaster). Presumably Exondys 51 was one of the treatments that Gabe’s family wanted to see made legally available.

Governor Bentley issued a proclamation in honor of Griffin on Monday proclaiming August 8, 2016 as Ride4Gabe Day to End Duchenne.

Hopefully today’s announcement will bring us closer to that end. The ‘Alabama Political Reporter’ extends prayers for Gabe and all Duchenne’s patients.

Congressman Gary Palmer represents Alabama’s Sixth Congressional District.

 

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