Alnylam Pharmaceuticals announced that it has completed patient enrollment for its Phase 3 HELIOS-A trial of vutrisiran, which will…
News
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000…
In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier.
Onpattro (patisiran) has been approved in Brazil to treat hereditary transthyretin amyloidosis in adults with stage 1 or stage 2 polyneuropathy,…
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few…
The European Commission (EC) has approved the once-daily oral medication Vyndaqel (tafamidis) for the treatment of cardiomyopathy — a disease of the…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening…
The V122I gene mutation, found primarily in people of African descent with hereditary transthyretin amyloidosis (ATTR), is associated with heart failure in individuals…
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had…
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