First new FDA-approved treatment option in nearly 20 years for families living with this rare lysosomal storage diseaseFirst FDA-approved ...

Lysosomal Storage Disorders are genetic conditions caused by enzyme deficiencies that lead to harmful substance buildup in cells, resulting in progressive organ damage. Common disorders in India ...

December 2, 2011 — Lysosomal storage disorders such as Fabry's disease and Pompe's disease are much more common than previously thought, particularly atypical later-onset forms, a new study suggests.

Denali Therapeutics' Avlayah is the first drug for neurological complications associated with the rare lysosomal storage ...

The FDA granted accelerated approval to tividenofusp alfa (Avlayah) for the treatment of mucopolysaccharidosis type II (MPS II), a rare disease also known as Hunter syndrome, the agency announced ...

The early funding will help transition its research into scalable therapies targeting lysosomal storage disorders.

In a major breakthrough in the fight against rare diseases, a Gujarat-based institute has established India’s first national ...

The FDA has approved Denali Therapeutics’ enzyme replacement therapy for a genetic lysosomal storage disease, breaking the agency’s recent string of rejections for rare disease treatments.

PARAMUS, NJ, Feb. 27, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal ...

Azafaros, a company aiming to become a leader in lysosomal storage disorders (LSDs), focused on addressing especially neurological symptoms, today announced the publication of proof-of-concept ...

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 ...

In a significant step towards tackling rare genetic disorders, a Gujarat-based institute has established India's first ...