Centogene has extended its partnership with Takeda to keep providing genetic testing services for the diagnosis of lysosomal…
Articles by Patricia Inácio, PhD
In previously untreated Fabry disease patients with heart involvement, 18 months of treatment with Galafold (migalastat) stabilized measures…
A long clinical history of cardiac alterations, including heart valve impairments, without an apparent cause and accompanied by kidney dysfunction…
Note: This story was updated Dec. 12, 2022, to correct the date by which the FDA must announce its…
Left ventricular hypertrophy (LVH) — a condition when the walls of the heart’s left pumping chamber (left ventricle) become thickened…
Protalix BioTherapeutics and its partner Chiesi Global Rare Diseases have applied to the European Medicines Agency (EMA) for the approval of PRX-102 (pegunigalsidase…
Lucerastat, an investigational substrate reduction therapy for Fabry disease, has failed to outperform placebo at reducing neuropathic pain in…
A Phase 1/2 clinical trial of Sangamo Therapeutics’ experimental gene therapy ST-920 in people with Fabry disease has…
The European Commission has expanded the use of Galafold (migalastat) to children starting at age 12 and weighing at…
In people with Fabry disease, starting enzyme replacement therapy (ERT) before the onset of chronic kidney disease —…