A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...
MedPage Today on MSN
Gene Therapy Shows Lasting Benefit for Children With Rare Disorder
For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...
Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.
Denali Therapeutics has become the latest on a growing list of biotechs to experience an FDA review delay in recent months. | ...
The Batten Disease pipeline presents opportunities with over 10 companies developing 12+ drugs in varied clinical stages. Novel approaches, including gene therapy and enzyme replacement, address unmet ...
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological ...
Children with Hunter Syndrome lack a certain enzyme in their blood. The Food and Drug Administration just approved an enzyme-replacement therapy for these patients. It had its start at UNC Hospitals.
Ottawa, Sept. 24, 2025 (GLOBE NEWSWIRE) -- The global enzyme replacement therapy market size was valued at USD 10.4 billion in 2024 and is predicted to hit around USD 24.71 billion by 2034, rising at ...
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