FDA grants orphan drug designation to UAB-developed gene therapy for ALS

by Universitat Autònoma de Barcelona (UAB)

11 months ago
Amyotrophic Lateral Sclerosis (ALS), viral vector of the AAV (adeno-associated virus), Klotho (s-KL) protein, orphan drug

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral Sclerosis (ALS) developed at the Universitat Autònoma de Barcelona and li...

A natural compound designated as an orphan drug for cystinuria

by Hospital Universitari de Bellvitge

5 years ago
Cystinuria, orphan drug, L-ergothioneine, rare disease

Cystinuria is a rare disease characterized by the formation of cystine stones in the kidney and urinary tract.

Minoryx Therapeutics receives Orphan Drug Designation from the US FDA for its lead candidate MIN-102

by Professional Newco

9 years ago
MIN-102, Adrenoleukodystrophy, X-ALD, Orphan drug

MIN-102 targets X-linked Adrenoleukodystrophy (X-ALD), a life threatening orphan CNS disease with high unmet medical need. Orphan drug status for MIN-102 has now been granted by both the FDA and the E...