Changes in Heart, Kidney Function May Be Undiagnosed Fabry Disease
A long clinical history of cardiac alterations, including heart valve impairments, without an apparent cause and accompanied by kidney dysfunction…
Articles by Patricia Inácio, PhD
FDA Will Give Potential Treatment PRX-102 Another Look
Note: This story was updated Dec. 12, 2022, to correct the date by which the FDA must announce its…
Heart Disease, Other Symptoms Could Be Sign of Late-onset Fabry
Left ventricular hypertrophy (LVH) — a condition when the walls of the heart’s left pumping chamber (left ventricle) become thickened…
Approval of PRX-102, Plant-based ERT for Adults, Sought for Europe
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 Fails to Lessen Neuropathic Pain in Phase 3 Trial
Lucerastat, an investigational substrate reduction therapy for Fabry disease, has failed to outperform placebo at reducing neuropathic pain in…
3rd Patient Group Being Enrolled in Trial of Fabry Gene Therapy ST-920
A Phase 1/2 clinical trial of Sangamo Therapeutics’ experimental gene therapy ST-920 in people with Fabry disease has…
Galafold Approved in EU for Children 12 and Older With Fabry
The European Commission has expanded the use of Galafold (migalastat) to children starting at age 12 and weighing at…
Starting ERT Before Kidney Disease Onset Leads to Better Outcomes
In people with Fabry disease, starting enzyme replacement therapy (ERT) before the onset of chronic kidney disease —…
Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
Quick Approval for COVID Vaccines a Concern, Rare Disease Patients Say
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…