Water – A refuge from Duchenne
I am sitting and watching my boy experiencing freedom. Free from his wheelchair, free from constriction, and tightness and free from Duchenne – for a moment. We are in Miami, basking in the warm and humid weather, and my son is floating in the water light as air and is soaring. The sun is beating […]
Duchenne Parent Guide for Sanity
It is not often that we find tools and resources for parents for coping and living with Duchenne. Each one of us is consumed in finding the best therapy for our boys, getting them into the right clinical and the best clinical care. NONE of us are focusing on our own needs. What? Do we […]
FDA Finally Approves First Ever Treatment Drug – Finally!
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 without bureaucratic “drama” and in-fighting. There were definite opponents for approval, based on the nonsense of […]