Clemson researchers believe they’re on the verge of a breakthrough treatment for Sanfilippo Syndrome, which is also known as ...

Ultragenyx Pharmaceutical RARE announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 ...

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 ...

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 ...

William Blair has initiated coverage on Denali Therapeutics Inc (NASDAQ:DNLI), a company focused on developing drugs for ...

Clark Willmore was recently diagnosed with Sanfilippo syndrome, a degenerative disease that causes similar symptoms to Alzheimer's but in children. James Dean, a lifelong Vol fan, saw a post on ...

Dr. Brennan’s current areas of research focus on speech, language, and communication in individuals with rare conditions, including Smith-Magenis Syndrome (SMS), Sanfilippo Syndrome, Autoimmune ...

based gene therapy for Sanfilippo syndrome type A (MPS IIIA), and ABO-101 (AAV NAGLU), an AAV based gene therapy for Sanfilippo syndrome type B (MPS IIIB). The company was founded in 1974 and is ...

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 ...

such as DNL126 for Sanfilippo syndrome type A. Most recently, Denali Therapeutics initiated dosing in a global BEACON Phase 2a trial of BIIB122 (DNL151) for LRRK2-associated Parkinson’s disease.