For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

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 ...

Denali Therapeutics has become the latest on a growing list of biotechs to experience an FDA review delay in recent months. | ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

Dublin, Oct. 02, 2025 (GLOBE NEWSWIRE) -- The "Mucopolysaccharidosis Market - A Global and Regional Analysis: Focus on Treatment, Disease Type, Route of Administration, End User, Country, and Region - ...

Amicus Therapeutics (FOLD) remains a Buy, driven by strong product momentum and new assets. Read here for more investment ...

Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy.

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 ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

(MENAFN- GlobeNewsWire - Nasdaq) Pompe disease is a rare genetic disorder resulting from GAA gene mutations, leading to acid alpha-glucosidase deficiency and glycogen buildup. Key symptoms include ...