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 ...

The raise will go toward trialing the company’s lead drug for phosphomannomutase-2 congenital disorder of glycosylation, a ...

We sought to evaluate whether treatment was safe and effective in patients with Pompe disease when alglucosidase alfa therapy was initiated after 6 months of age. The present study was an open ...

Briefly, patients must have had documented symptoms of infantile-onset Pompe disease and been <7 mo old at enrollment. Patients with respiratory insufficiency, heart failure, or any prior ...

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 ...

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 ...

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.

Also read: Family shares story of newborn's heart surgery success thanks to BBCH care Mckenzie has infantile Pompe disease. She's the only child in the state with this genetic disorder.

Moving on to Pompe disease, 5000–10000 people are globally diagnosed with it every year. The main cause of fatality in these patients is respiratory failure. Globally, $1.5bn is spent on ERT ...

Combined with newborn screening for both infantile- and late-onset Pompe disease, GENV-002 can be administered to all patients as infants, a decade or more in advance of non-genome editing AAV ...