Clemson researchers conducting breakthrough research on childhood Alzheimer’s
Clemson researchers believe they’re on the verge of a breakthrough treatment for Sanfilippo Syndrome, which is also known as ...
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RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy
Ultragenyx Pharmaceutical RARE announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 ...
Denali Therapeutics Neurodegenerative Disease Platform Sparks Analyst Optimistic
William Blair has initiated coverage on Denali Therapeutics Inc (NASDAQ:DNLI), a company focused on developing drugs for ...
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College football fans unite to raise thousands for young Ohio State fan with rare disease
It was revealed that Clark had Sanfilippo syndrome, a rare disease that is often described as childhood Alzheimers. "In my mind, I'm sitting there thinking, like, I'm not going to watch him grow ...
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Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
About UX111 UX111 is a novel in vivo gene therapy in Phase 1/2/3 development for Sanfilippo syndrome type A (MPS IIIA), a rare fatal lysosomal storage disease with no approved treatment that ...
Denali Therapeutics Maintains Strong Buy Rating Amid Setbacks, Fueled by Promising BBB Platform
Stifel Nicolaus analyst Paul Matteis has maintained their bullish stance on DNLI stock, giving a Buy rating on January 3.Don't Miss Our New ...
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Clemson researchers conducting breakthrough research on childhood Alzheimer’s
GREENVILLE, S.C. (FOX Carolina) - Clemson researchers believe they’re on the verge of a breakthrough treatment for Sanfilippo Syndrome, which is also known as childhood Alzheimer’s.
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Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). “The path to get a treatment to the point of a BLA filing has been long and perilous for the Sanfilippo ...