Blog
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...
FDA – NOT FOR DUCHENNE
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...
Clinical Trial – Day 1
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...
Two classmates – two trials….heroes amongst us
Today I had a sad day visiting a classmate of Alexander’s at Children’s Hospital. He had a malignant brain tumor removed 6 days ago. His name is Cooper and he is 9. His mom is a friend of mine, who recently found out...
Day at the Clinic
Today was the Center for Duchenne Muscular Dystrophy clinic day… A completely dreaded day for us all. How exhausting both emotionally and physically. The first thing I must say is the fact that Alex did NOT want to...
CLINICAL TRIAL – No easy choices
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...
