Soon after, Logan was diagnosed with Sanfilippo syndrome. As Logan’s mom, Noelle, described: “Sanfilippo syndrome is a ...

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 ...

Denali Therapeutics DNLI has performed well in the past 12 months amid a volatile market. Shares of the company have gained ...

A young Ohio State fan has brought together Buckeyes and Volunteers fans alike in a heartwarming effort to support his family ...

(NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV ...

New Delhi, Jul 07 (ANI): Hollywood celebrities helped raise USD 1 million to help a child get treatment for the fatal disease Sanfilippo syndrome. A family of four had launched a GoFundMe page ...

(RARE), announced on Thursday that it has submitted a Biologics License Application to the U.S. FDA for UX111, an AAV gene therapy for Sanfilippo Syndrome Type A. Sanfilippo Syndrome Type A ...

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 showcases its BBB platform at J.P. Morgan Conference, highlighting DNL310's Hunter syndrome progress and plans for commercialization.

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 ...

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 ...