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…
News
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…
While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove…
The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to Alnylam Pharmaceuticals’ vutrisiran as a potential treatment of adults with …
Sudoscan, a device that evaluates sweat gland function, may help physicians improve the diagnosis of familial amyloid polyneuropathy — distinguishing these patients from…
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