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.

Elevidys was approved for a few types of patients, but since a couple of patients in the trial treatment died of acute liver failure afterwards, the FDA halted access to those in wheelchairs like her son, who lost his ability to walk at age 9. Sarepta Therapeutics said those severe outcomes were not the result of the treatment. The two DMD patients who died were in advanced stages, and a third fatality occurred in a separate trial of a higher-dose gene therapy for a different muscle disease.

Their son has staggering medical costs, including a $30,000 power wheelchair and steroid medications that average $30,000 per month. The Muscular Dystrophy Association used to cover the costs that insurance did not pick........

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