The European Medicines Agency (EMA) has agreed to review Alnylam Pharmaceuticals’ application requesting the approval of vutrisiran, its second-generation RNA interference (RNAi) therapy candidate…
News
A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community…
Vutrisiran, an investigational therapy for familial amyloid polyneuropathy (FAP), eased neurological impairments, lessened heart stress, and ameliorated quality of life, nutritional status, and…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which…
Long-term treatment with Tegsedi (inotersen) helps preserve quality of life in people with familial amyloid polyneuropathy (FAP) for up to three years, according…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for…
Changes in the function of the retina, in combination with nerve damage in the cornea, can be used as early biomarkers of hereditary transthyretin (hATTR)…
Registration is now open for Global Genes‘Â 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and…
Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also…
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