Onlymyhealth11 天
What Is Sanfilippo Syndrome: Symptoms, Treatment, and the Impact on Children’s Lives
For some families, this is a daily reality due to rare genetic disorders. One such condition is Sanfilippo syndrome, which creates significant hurdles for affected children and their families.
Bored Panda on MSN4 个月
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 ...
来自MSN1 个月
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 ...
Nasdaq1 个月
Ultragenyx Submits BLA To FDA For UX111 Gene Therapy For Sanfilippo Syndrome Type A
(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 ...