A U.S.-based Phase 1/2a clinical trial testing AMT-191, uniQure’s gene therapy treatment for Fabry disease, has completed enrollment…
Articles by Patricia Inácio, PhD
The first patient has been dosed in uniQure’s U.S. Phase 1/2a clinical trial of AMT-191, its gene therapy candidate…
A woman in her 20s with Fabry disease was safely treated with agalsidase alfa — a commonly used…
A man with Fabry disease experienced an easing of neuropathic pain, or nerve pain, with medical cannabis after failing…
Incorporating a three-tiered approach to newborn screening (NBS) makes it highly feasible for large-scale programs, according to a recent study…
A 55-year-old man with a Fabry disease-related tumor in the large intestine was treated successfully with a nonsurgical procedure…
Fabry disease remains widely underdiagnosed, especially among women, according to a new population-based study that identified one undiagnosed case…
Patients prefer receiving enzyme replacement therapy (ERT) at home, citing savings in travel time and cost, over clinical visits,…
Centogene has extended its partnership with Takeda to keep providing genetic testing services for the diagnosis of lysosomal…
In previously untreated Fabry disease patients with heart involvement, 18 months of treatment with Galafold (migalastat) stabilized measures…