Hosted on MSN14d
First patient dosed in Phase Ib Pompe disease trial
The Phase Ib trial is set to test a gene therapy designed to target a rare disorder that causes muscle and respiratory failure.
TheBetterIndia21h
‘No One Should Face This’: After Losing Daughter, Dad Ensures No Rare Disease Patient Suffers Alone
Through policy framework shifts, appeals to the court for rare disease funds, and starting a centre in Bengaluru, Prasanna ...
precisionmedicineonline21h
FDA Clears Aro Biotherapeutics' siRNA Drug IND for Late-Onset Pompe Disease
The company has already kicked off an open-label Phase I trial of ABX1100 in Canada following positive results from a study of healthy volunteers.
India Today8d
Rare disease patients in crisis as delays stall national policy implementation
Patient advocacy groups warned that delays in implementing India's rare disease policy have left many, including children, in ...
PharmiWeb7d
Amicus Therapeutics launches Amicus Ignite, a proactive community funding programme for initiatives that support people living with Pompe disease
Amicus Therapeutics UK Limited (Nasdaq: FOLD), a patient-dedicated biotechnology company, today announced the launch of Amicus Ignite, a proactive community funding programme to support initiatives ...
Aro Biotherapeutics Announces U.S. FDA Clearance of IND Application for ABX1100 for Potential Treatment of Late-Onset Pompe Disease (LOPD)
Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced that it has received U.S. Food and Drug Administration (FDA) ...
Amicus Therapeutics Announces Presentations and Posters at the 21st Annual WORLDSymposium™ 2025
PRINCETON, N.J., Feb. 03, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that two oral presentations and 20 posters across its development programs will be included at ...