Levels of neurofilament light chain (NfL) — a proposed biomarker of several neurological disorders — are significantly higher in people with…
News
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the…
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July…
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based…
Long-term treatment with Vyndaqel (tafamidis) may reduce the risk of death in people with familial amyloid polyneuropathy (FAP), a study shows. The…
The progressive nature of hereditary transthyretin amyloidosis polyneuropathy (hATTR-PN), also known as familial amyloid polyneuropathy (FAP), limits patients’ health-related quality of life across all…
Alnylam Pharmaceuticals announced that it has completed patient enrollment for its Phase 3 HELIOS-A trial of vutrisiran, which will…
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.
Recent Posts
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- Early diagnosis, treatment improves survival in ATTR-CM: Study
- FDA lifts clinical hold on Phase 3 trial of nex-z for hATTR-PN