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…
News
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…
Levels of neurofilament light chain (NfL) — a proposed biomarker of several neurological disorders — are significantly higher in people with…
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…
Recent Posts
- Amvuttra may ease symptoms and improve daily life in ATTR-CM
- Early nerve damage seen in woman with rare TTR mutation
- AInylam initiatives aim to speed ATTR-CM diagnosis, coordinate care
- Blood test for Alzheimer’s may help in making hATTR-PN diagnosis
- Women with ATTR-CM face gaps in treatment, new study reports