Audentes Pompe Gene Therapy Trial News.
Audentes is now enrolling patients with late-onset Pompe disease in the Phase 1/2 clinical trial.
Audentes is now enrolling patients with late-onset Pompe disease in the Phase 1/2 clinical trial.
New born screening has long been championed by AGSD-UK. Now you have an opportunity to support a larger national campaign.
A trial of a new treatment for GSD2 has been halted. VAL-1221 was delivered intravenously in late-onset Pompe patients.
We mark the passing of Dr Clive Tonks in his mid-eighties. He was a past trustee and committee member of AGSD-UK.
Ismail lives with Infantile Onset Pompe Disease. Aged 19, his abiding passion is football. He plays using an adapted wheelchair.
A new series from Netflix, in which a doctor crowd-sources diagnoses for mysterious medical conditions, opens with a story mentioning GSD.