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.

Please provide your email address to receive an email when new articles are posted on . An Italian pharmaceutical company and a Boston-area biotechnology research firm announced a partnership to ...

Azafaros, a company building a portfolio to become a leader in lysosomal storage disorders with the goal of addressing neurological symptoms, today announced that it will present at J.P. Morgan’s 44th ...

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE: 4552) made significant contributions at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024, held ...

Cholesteryl Ester Storage Disease (CESD) is a rare autosomal recessive multisystem condition that belongs to the lysosomal storage disorders family. The liver is one of the most common sites of ...

Alzheimer's disease (AD) currently has no cure and is predicted to affect over 100 million people worldwide by 2050. Ongoing research is focused on two key neurotoxic proteins: amyloid beta (Aβ) and ...

ST. LOUIS, Mo., April 09, 2025 (GLOBE NEWSWIRE) -- M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies ...

Patients with Niemann-Pick disease type C who received N-acetyl-L-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, showed reduced neurologic signs and ...