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Targeted Therapies Aim at the Root of the Disease At the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in Dallas, Dr. Matthew Alexander, associate professor of pediatric neurology and genetics at the University of Alabama, Birmingham, led a...
100 days after the FDA's legally mandated decision date,the FDA announced accelerated approval for Eteplirsen by Sarepta Therapeutics. Eteplirsen is the first therapy drug approved for Duchenne which works to skip over the deleted exons. The approval did come...
April 26, 2016 As I sit bleary-eyed on the airplane flying home from what should have been a “successful” trip to DC, I am still stunned and disheartened from yesterday after a grueling TWELVE HOUR meeting with the FDA and it’s Advisory Committee, that went...
I guess you can say - "we are lucky.' After 5 days of putting Alexander through extensive testing (a 3 hour MRI, extensive bloodwork, ECG, Pulmonary, 2 days of physical therapy assessment, fecal testing (ek!) and a physical exam), we received official word last...
On a Saturday morning at 9:00am I received a call from our Neurologist at UCLA. He called to tell me the news that there is a clinical trial at UCLA that Alexander could qualify for. After a 45 min conversation, I was left hopeful but at the same time anxious and a...
