NORD’s Advice on COVID-19: You’re Stronger Than You Think
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 ... Read more
Updates
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NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because ... Read more -
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
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 ... Read more -
Paying Attention to Mental Health During Social Isolation
The COVID-19 pandemic has surged to unprecedented levels. Since last week’s column, many schools, workplaces, and public gatherings have been placed on hold. For ... Read more -
Long-term Vyndaqel Improves Survival in FAP, Study Suggests
Long-term treatment with Vyndaqel (tafamidis) may reduce the risk of death in people with familial amyloid polyneuropathy (FAP), a study shows. The study, “Evaluation ... Read more
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Being Prepared: Neuropathy Patients, Social Distancing, and the Coronavirus
By now the virus that causes COVID-19 has expanded to numerous countries, causing a ... -
How Neuropathy Patients Can Cope with a Diagnosis
The presence of peripheral neuropathy symptoms such as numbness, tingling, and burning sensations in ... -
Alnylam Completes Enrollment for Phase 3 HELIOS-A Trial Testing Vutrisiran
Alnylam Pharmaceuticals announced that it has completed patient enrollment for its Phase 3 HELIOS-A ... -
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media ...
Tafamidis Delays Disease Progression in Patients with Transthyretin Amyloid Polyneuropathy, Pfizer Study Suggests
News
An interim analysis of the Phase 3 trial for tafamidis (Fx-1006A) as a therapy for patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP) shows the investigational treatment delays disease progression, Pfizer announced. The study, “Long-term safety and efficacy of tafamidis for ... Read more