The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization…
News
Five advocacy groups for familial amyloid polyneuropathy (FAP) are sharing a total $270,000 award given by…
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting…
Tegsedi (inotersen) continued to slow disease progression and improve the quality of life in people with familial…
The Portuguese Medicine Regulatory Authority, known as Infarmed, has added Tegsedi (inotersen) — an approved treatment for stage 1 or 2 polyneuropathy…
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…
Recent Posts
- New analyses back Amvuttra as first-line treatment for complex heart cases
- Injection therapy Wainua offers ‘ray of hope’ for hATTR-PN patients
- Understanding the patient’s perspective in a fast-paced world
- Attruby may reduce outpatient heart failure worsening in ATTR-CM
- Being a caregiver means becoming an expert on your loved one’s disease