Source: Town Hall
by Rachel Alexander
“The U.S. Senate Special Committee on Aging conducted a hearing on February 26 to investigate why the FDA is holding up the approval of desperately needed new drugs for those with rare diseases. Many of the people suffering with these ailments are children with short life expectancies. Those with Duchenne Muscular Dystrophy (DMD), which is progressive muscular deterioration, end up in a wheelchair at about age 11-12, and die in their 20s. Angelina Olivera, the mother of a 14-year-old son who has DMD, who also watched her brothers die at ages 20 and 22 of the disease, told me there is only one promising new treatment for DMD but the FDA won’t approve it for her son Ryu. A study was conducted on 1,200 patients using Elevidys from Sarepta Therapeutics and revealed promising results. The gene therapy slowed disease progression by 70-73 percent compared to untreated children.” (03/02/26)