Alabama family teams up with Shelby and Palmer to fight rare muscular disease

WASHINGTON — Earlier this year year Scott and Traci Griffin whose 10 year-old-son, Gabe, suffers from an extremely rare form of Muscular Dystrophy, made history when they successfully lobbied for “Gabe’s Right to Try Law.” Now the family is teaming up with Senator Richard Shelby (R-AL) and Congressman Gary Palmer (R-AL6) to ensure the family gets to take their ideas for patients in a similar situation all the way to the FDA.

“Gabe’s Right to Try Law” allows new medications already in the human trial portion of FDA approval to be tried by Alabama patients with terminal, life-threatening illnesses. There is currently no nationwide equivalent.

Congress has tasked the FDA with considering views from the patient community in assessing the risks and benefits of proposed new drugs designed to treat rare and deadly conditions for which no current treatment options exist, but the Griffins believe the agency still takes too much time and is too selective in their trial process to be of much hope to Gabe and other children in his position.

Caused by an absence of a protein that helps keep muscles intact, Duchenne’s disease is a fatal disorder occurs during early childhood and causes weakness and muscle wasting that only worsens over time. There is currently no cure, but Gabe’s family believe that breakthroughs are just around the corner.

Both Sen. Shelby and Rep. Palmer sent FDA commissioner Dr. Stephen Ostroff letters urging him to meet with the Griffins.

“Sadly, Gabe’s muscles will continue to deteriorate until he can’t walk, breathe or eat by himself,” Sen. Shelby wrote. “Without access to new treatment, these devastating effects of Duchenne will impact Gabe well before he turns twenty years old.”

“Gabe is currently prescribed FDA-approved steroids with side effects of stunted growth, extreme sensitivity to light, postponed puberty, and brittle bones. However these drugs do nothing to target the underlying problem, a missing protein, which causes Duchenne.”

“We would also appreciate you scheduling a meeting with Gabe and his parents, as
representatives of the Duchenne patient community, in Washington at a mutually convenient time in the future,” wrote Rep. Palmer.

Scott Griffin said he is glad to have his elected representatives on his son’s side.

“We are blessed with elected officials who understand that time is unfortunately not on our side in our effort to save Gabe and the other children dying from Duchenne,” said Mr. Griffin.

Last year to raise awareness of Gabe’s condition and the importance of right-to-try legislation, the Griffin family’s Hope 4 Gabe foundation organized a 3,400 mile bike ride across the country with Alabamian Michael Staley, former chief of staff to Republican former Congressman Spencer Bachus.