FDA Approves First Gene Therapy for Children with MLD & More — This Week in RARE Daily

March 21, 2024

This Week in RARE Daily is a feature from Global Genes where you can get a quick rundown of the top 5 headlines in the rare disease space from our editorial staff. Here are top stories from this past week, March 15-21, 2024:

FDA Approves First Gene Therapy for Children with MLD
Orchard Therapeutics’ Lenmeldy is now the first FDA-approved gene therapy for the rare and fatal condition metachromatic leukodystrophy.

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Congressman Proposes Legislation to Combat Rare Diseases Impacting Women
Congressman from New Jersey to help fund efforts to increase the number of women in clinical research trials for rare diseases and blood disorders.

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Experimental Gene Therapy for GAN Shows Promise in NIH Trial
NIH trial for experimental gene therapy for the rare neurodegenerative disease giant axonal neuropathy shows promise.

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Florida Passes Bill to Establish Rare Disease Research Grant Program
Florida legislature establishes The Andrew John Anderson Pediatric Rare Disease Grant Program, named for the son of Republican State Representative Adam Anderson, whose son had Tay-Sachs disease

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EveryLife, Backed by Amgen, Offers Scholarships for College Students with Rare Diseases
Everylife Foundation for Rare Diseases and Amgen join to offer 88 $5,000 scholarships for college students with rare diseases for the fifth year.

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