MyChesCo on MSN

Experimental Pompe therapy shows early signal ahead of key San Diego reveal

PHILADELPHIA, PA — Aro Biotherapeutics said it will present interim clinical data next month from its ongoing Phase 1b trial ...
Business Insider

Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study of AT-GAA at the 24th International Annual Congress of the World Muscle Society ...

Persistent and Durable Responses Across Safety, Functional Outcomes and Biomarkers for ERT-Naïve and ERT-Switch Patients in Cohorts 1, 2 and 3 Out to Month 24 Data in Additional Switch Patients ...
MedCity News

Sanofi Finds Way to Maze Therapeutics to Get Its Next Pompe Disease Drug

Sanofi has two therapies for the rare disorder Pompe disease, one of which is a relatively recent entry into the market. But a drug candidate from Maze Therapeutics would offer advantages over both ...
Business Insider

Dyne Therapeutics Presents New Preclinical Data Demonstrating the Potential of the FORCE™ Platform to Deliver Enzyme Replacement Therapy to Muscle and CNS in Pompe Disease

WALTHAM, Mass., June 24, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for ...
EurekAlert!

Genethon signs licensing agreement with AskBio for the development of an investigational gene therapy for Pompe disease

Genethon, a leading laboratory in gene therapy for rare diseases, today announced that it has entered into an exclusive, worldwide licensing agreement with AskBio, a gene therapy company wholly owned ...
WebMD

Managing Late-Onset Pompe Disease With Opfolda

Opfolda (miglustat) is a medicine taken with another medicine for certain people with late-onset Pompe disease. Pompe disease is a rare genetic disorder that is caused by an abnormal gene for a ...

Belief BioMed Congratulates Partner AskBio on IND Acceptance by FDA for Investigational Gene Therapy for Late-Onset Pompe Disease

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, recently announced that the United States Food and Drug Administration (FDA) has ...