Bored Panda on MSN3mon
This Family Advocates For Rare Sanfilippo Syndrome In Hopes That Others Have Better Chances
Soon after, Logan was diagnosed with Sanfilippo syndrome. As Logan’s mom, Noelle, described: “Sanfilippo syndrome is a ...
Nasdaq1mon
RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy
AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway. Earlier this year, the FDA aligned with Ultragenyx, after a thorough ...
Fox Carolina17d
Clemson researchers conducting breakthrough research on childhood Alzheimer’s
Researchers have identified four drugs already on the market and are testing them to see if they work against Sanfilippo syndrome. “They are approved for something else currently, but they are ...
Nasdaq1mon
Ultragenyx Submits BLA To FDA For UX111 Gene Therapy For Sanfilippo Syndrome Type A
If approved, UX111 would be the first approved therapy in the U.S. for Sanfilippo Syndrome Type A. RARE closed Wednesday's (Dec.18.2024) trading at $42.94, down 4.47%. In premarket trading ...
WTOL1mon
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 ...