For example, the drug miglustat (Zavesca) treats one form of Gaucher disease. Stem cell transplant uses donated cells to help the body make the missing enzyme. Treatments to manage symptoms of the ...

Johnson & Johnson’s Zavesca (miglustat), once an early oral alternative, is now a backup option due to tolerability concerns and CNS-related side effects. While these therapies control systemic ...

At present, no curative therapy is available for NPC. Treatment focuses on symptom management and is carried out by a multidisciplinary team. Currently, miglustat, (Zavesca™; Actelion Pharmaceuticals ...

Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) Now Approved in Japan for Adults with Late-onset Pompe Disease (LOPD)PRINCETON, N.J., and TOKYO, June 25, 2025 (GLOBE NEWSWIRE) -- Amicus ...

Amicus Therapeutics Announces Approval of Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) in Japan Provided by GlobeNewswire Jun 25, 2025, 11:00:00 AM ...

Amicus Therapeutics has released a post-hoc analysis from the PROPEL study, published in Muscle and Nerve, which evaluated the efficacy of cipaglucosidase alfa-atga combined with miglustat (cipa ...

New Analysis of Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) Published in Muscle and Nerve Amicus Therapeutics, Inc. Tue, Jun 3, 2025, 7:00 AM 7 min read ...

Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) Now Approved in Japan for Adults with Late-onset Pompe Disease (LOPD)PRINCETON, N. Tuesday, 02 January 2024 12:17 GMT عربي ...

Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) Now Approved in Japan for Adults with Late-onset Pompe Disease (LOPD)PRINCETON, N.J., and TOKYO, June 25, 2025 (GLOBE NEWSWIRE) -- Amicus ...

Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) Now Approved in Japan for Adults with Late-onset Pompe Disease (LOPD) PRINCETON, N.J., and TOKYO, June 25, 2025 (GLOBE NEWSWIRE) -- Amicus ...

PRINCETON, N.J., June 03, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced the publication of a post-hoc analysis of data from the ERT-experienced cohort of the PROPEL ...

Amicus Therapeutics Inc. (NASDAQ:FOLD) is one of the best biotech stocks to invest in now.On June 3, Amicus Therapeutics announced the publication of a post-hoc analysis of data from the ERT ...