Two European Bio-Tech companies are investigating the suitability of combining the two companies’ technology into one powerful gene therapy.

Patients with a genetic disease, adrenoleukodystrophy, or ALD, causing brain cell death were successfully treated with gene therapy.

Using CRISPR, researchers have successfully treated congenital muscular dystrophy type 1A (MDC1A), a rare disease that can lead to severe mu

COMMAD is a syndrome which presents, ocular holes, dense bones, small eyes, large head, albinism, and deafness. A rare example of a disease

123Genetix (www.123genetix.com) is developing a big data/deep learning solution to assist researchers: Develop new hypotheses; Design and ru

PTC Therapeutics (NASDAQ: PTCT) is a global biopharmaceutical company focused on the discovery, development, and commercialization of novel

When the Bragg family learned their daughter, Clara had GM1, a rare Lysosomal Storage Disease they, like many parents with children with rar

Research carried out on a Niemann-Pick type C1 (NPC1) treatment can lead to a new generation of potential therapies for NPC1 and other simil

Alexion Pharmaceuticals, Inc. presented new data displaying benefits of Soliris (eculizumab) treatment for patients with PNH, regardless of

Takeda and Seattle Genetics announced promising results from its Phase 3 ECHELON-1 Clinical Trial that evaluated Adcetris in Frontline Advan