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ALS 'Compassionate Use' Again Hits National Media


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When Neil Shneider, MD, PhD, got results back that Lisa Stockman Mauriello s amyotrophic lateral sclerosis (ALS) was driven by a
SOD1 mutation, enrollment in the VALOR study had just closed.
The phase III trial is testing Biogen s investigational antisense oligonucleotide (ASO) aimed at knocking down levels of the mutant protein thought to play a major role in contributing to ALS in the 2% of patients with
SOD1 mutations.
Shneider, an investigator on the VALOR trial at Columbia University who has long worked on therapies targeted to specific mutations in ALS, figured he d be able to go the route of expanded access (also called compassionate use ) instead. He requested access from Biogen for the 51-year-old mother of three on Feb. 4. ....

New Haven , United States , Jaci Hermstad , Bob Mauriello , Stockman Mauriello , Lisa Stockman Mauriello , Kristina Fiore , Daniel Mcintyre , Barlett Steele , Alfred Sandrock , Neil Shneider , Gregg Gonsalves , Syneos Health , Yale School Of Public Health , Early Access Program , Columbia University , New England Journal , Medpage Today , Yale School , Public Health , Timmerman Report , புதியது புகலிடம் , ஒன்றுபட்டது மாநிலங்களில் , பாப் ம Ri ரியல்லோ , லிசா ஸ்டாக்மந் ம Ri ரியல்லோ , கிறிஸ்டினா ஃபியரி ,