The Indian Medical Parliamentarians Forum (IMPF) - a cross-party group of 45 MPs who are also medical professionals -- has sought uninterrupted access to Enzyme Replacement Therapy for children ...

(Getty Images) A connection is increasingly evident between lysosomal dysfunction and age-associated illnesses such as Parkinson and Alzheimer diseases. The lysosome is emerging as a shared axis of ...

A Lecture / Workshop on depression and bipolar disorder featuring three Psychiatrists from the University of South Florida School of Psychiatry and Behavioral Neuroscience presented by DBS Tampa Bay, ...

The FDA on Friday approved chenodiol (Ctexli) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Chenodiol -- a naturally occurring human bile acid also known as chenodeoxycholic ...

Glycogen storage disease (GSD) refers to a group of rare genetic conditions that affect how the body stores and breaks down glycogen. The most common type is type I, also called von Gierke disease.

While it was just last week that Zevra Therapeutics’ Miplyffa became the first FDA-approved treatment for the lysosomal storage disorder Niemann-Pick disease type C (NPC), IntraBio has already ...

For Zevra Therapeutics and its rare neurodegenerative disease med arimoclomol, the second time’s the charm. Friday, the FDA approved Zevra’s arimoclomol capsules to treat the rare lysosomal storage ...

Rocket Pharmaceuticals, Inc. (RCKT) announced that it had been able to complete enrollment for its pivotal phase 2 study using its gene therapy RP-A501 for the treatment of male patients with Danon ...

Ultragenyx is gearing up to take its glycogen storage disease (GSD) treatment to regulators next year after the gene therapy helped patients reduce their intake of cornstarch, which is currently used ...