The Portuguese Medicine Regulatory Authority, known as Infarmed, has added Tegsedi (inotersen) — an approved treatment for stage 1 or 2 polyneuropathy…
News
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in…
Treatment with Onpattro (patisiran) improves multiple aspects of quality of life in people with familial amyloid polyneuropathy (FAP), findings from a Phase 3…
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online…
Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and…
Spain’s Ministry of Health has added Tegsedi (inotersen), an injection treatment for stage 1 or 2 polyneuropathy in adults with familial amyloid polyneuropathy…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together…
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people…
A type of surgery that involves removal of the eye’s “gel” can improve vision in people with familial amyloid polyneuropathy (FAP), a new study…
FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect…
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