EL PASO, TEXAS (KFOX14\/CBS4) \u2014 An El Paso mother told a U.S. Senate hearing today that the FDA\u2019s decision to block some treatments for rare diseases has made it harder for families like hers to care for their children.<\/p>\n
Angelina Olivera, whose son has Duchenne muscular dystrophy, said she relies on medications approved by the FDA because the terminal illness has no treatment. <\/p>\n
Duchenne muscular dystrophy is a rare genetic disorder that causes muscle weakness and damage due to mutations in the dystrophin gene, which is essential for keeping muscle cells intact.<\/p>\n
In an interview with KFOX14\/CBS4, Olivera said an approved medication that was already in use has been stopped by the FDA, and that assistance from the Muscular Dystrophy Association has also stopped.<\/p>\n
\u201cBy blocking treatment, the FDA has made our lives so much harder,\u201d Olivera said. \u201cMy son is 14 years old. He could die tomorrow and the FDA seems to be more focused on bureaucracy versus the patient’s lives.\u201d<\/p>\n
At Thursday\u2019s Senate hearing, senators and experts also raised concerns about the administration\u2019s oversight of rare diseases, citing claims of inconsistent review processes.<\/p>\n
The hearing comes after Sarepta Therapeutics said it would pause all shipments of its gene therapy Elevidys for muscular dystrophy last July. <\/p>\n
Elevidys is the first gene therapy approved in the U.S for the disease, but has been under scrutiny after two teenage boys died last year from acute liver injury, a known side effect of the treatment. <\/p>\n
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