The Phase Ib trial is set to test a gene therapy designed to target a rare disorder that causes muscle and respiratory failure.

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 ...

Her mother, Kelsey, living in Espanola, about 40 minutes west of Sudbury, welcomes all the activity because Iris has a rare, ...

The offerings reflect an increase in IPO activity that could continue, as four other biotechs recently outlined plans to go ...

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder. The US regulator has approved ...

With mutations in the gene that creates the enzyme known as acid alpha-glucosidase (GAA), which the body utilises to break down glycogen (a stored form of sugar used for energy), Pompe disease is ...

Amicus Therapeutics could be just weeks away from getting EU approval for its new Pompe disease therapy Pombiliti after getting a positive opinion on the drug from the EMA's human medicines committee.

The government may slash duties on imported rare disease and cancer drugs in the Union budget aiming to make lifesaving ...

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 ...