Aro Biotherapeutics has dosed the first patient in a Phase Ib trial of ABX1100, a novel treatment for late-onset Pompe disease (LOPD), a rare inherited disorder that causes progressive muscle ...

PORTLAND, Maine (WGME) -- MaineHealth Barbara Bush Children's Hospital is crucial to so many families, some of which are ...

Pompe disease treatment market grows with advances in ERT and gene therapy, improving patient outcomes and access.

The US regulator has approved Nexviazyme (avalglucosidase alfa) for the treatment of patients aged one year of age and older with late-onset Pompe disease, which progressively attacks the heart ...

GAA is responsible for metabolising glycogen, which builds up in Pompe disease patients and causes progressive muscular degeneration. The initial readout will be a relief to Astellas, as AT845 ...

MZE829 Phase 2 HORIZON Trial Enrolling Patients with APOL1 Kidney Disease (AKD); Initial Data Expected in Q1 2026MZE782 Phase 1 Healthy Volunteer ...

Patients suffering from rare diseases are facing severe difficulties due to delays in receiving timely financial aid and ...

Megan’s affliction, Pompe disease, is exceedingly rare. The course of the disease varies by patient, but it causes children’s muscles to weaken and, eventually, their lungs may fail.

Around 1,000 patients with rare diseases are registered with Centres of Excellence in India. The government allocated ...

Prevalence of genetic diseases in ME/CFS patients How common are genetic diseases in children ... to take part in a research study which will tell us whether late-onset Pompe disease or Limb Girdle ...