Metachromatic Leukodystrophy

May 21, 2025

Lysosomal Storage Disorders, or LSDs, are rare genetic conditions that may not make headlines, but they affect thousands worldwide, often with life-changing consequences. LSDs happen when the body’s lysosomes, tiny cellular recycling centers, fail to break down waste materials due to missing or faulty enzymes. This...

Mar 19, 2024

Bristol Myers Squibb Enhances Neuroscience Arm with Karuna Therapeutics Buyout Bristol Myers Squibb has declared the finalization of its purchase of Karuna Therapeutics, Inc. With this acquisition concluded Karuna’s shares are no longer being traded on the Nasdaq Global Select Market, as Karuna is now fully owne...

Sep 25, 2023

Orchard Therapeutics’ Biologics License Application for OTL-200, a gene therapy under investigation for metachromatic leukodystrophy treatment, has been accepted by the FDA. This rare disease treatment received approval in Europe back in 2020. Notably, the FDA has granted Orchard’s application Priority Review statu...

Feb 08, 2022

Biogen's Aduhelm Marketing and Approval are Under Scrutiny in New FTC and SEC Investigations Biogen can't seem to get a break when it comes to the Alzheimer's disease drug Aduhelm. Along with limited sales and a constraining Medicare coverage plan, the medicine is now facing additional scrutiny as part of a pair...

Oct 23, 2017

Metachromatic Leukodystrophy (MLD) is a rare lysosomal storage disease which is genetic, degenerative, neurometabolic in nature. It is generally inherited from carrier parents and at present doesn’t have any cure. Patients suffering from MLD are deficient in the arylsulfatase-A enzyme, which is responsible for break...