Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder. The US regulator has approved ...

and preclinical programs for facioscapulohumeral muscular dystrophy and Pompe disease. Ocean Biomedical is a biopharma company that says it “accelerates the development and commercialization of ...

It offers oral therapy for adults with Fabry disease, and a unique two-part treatment designed for adults with late-onset Pompe disease. FOLD ranks 3rd on our list of the best Guru stocks.

Apr. 22, 2025 — Researchers have identified a potential new way to treat idiopathic pulmonary fibrosis (IPF), a deadly and currently incurable lung disease that affects more than 3 million people ...

Adding to the challenge is the disease’s rarity. ALS affects only two to three people per 100,000 annually, and there's a lack of awareness among general practitioners. “Most family doctors will ...

Nonalcoholic fatty liver disease (NAFLD) can be reversed with several lifestyle changes, including diet and physical activity. NAFLD is a condition where fat builds up around the liver and is ...

Chronic kidney disease (CKD) is defined as a progressive loss of renal function that lasts for more than 3 months, and is classified according to the degree of kidney damage – measured by the le ...

Inflammatory bowel disease is a chronic condition of unknown origin characterized by severe inflammation and mucosal destruction in the intestine. There are two main forms: Crohn s disease ...

AB-1002 is one of several clinical-stage gene therapy programmes being run by AskBio, along with candidates for Pompe disease, Parkinson’s disease, Huntington’s disease, multiple system ...