Preclinical study demonstrates improved gene therapy approach to treating a rare and devastating disease in children
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
The American Journal of Managed Care26d
5 Rare Diseases That Now Have Their First FDA-Approved Treatments
5. Metachromatic Leukodystrophy: Arsa-cel (Lenmeldy) Following priority review, orphan drug, rare pediatric disease, and regenerative medicine advanced therapy approvals, pediatric patients with ...
The Victoria Advocate15d
Children's Hospital of Philadelphia Preclinical Study Demonstrates Improved Gene Therapy Approach to Treating Metachromatic Leukodystrophy
In MLD, patients lack a properly functioning arylsulfatase A (ARSA) enzyme ... this progressive disease causes issues with movement and speech. Most children eventually lose the ability to ...
pharmaphorum3y
NICE rejects Orchard's gene therapy for rare childhood disease MLD
Orchard Therapeutics' gene therapy for rare childhood disease metachromatic leukodystrophy ... disorder caused by a deficiency in the ARSA gene coding for the enzyme arylsulfatase-A, which ...