News

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the…

Patient enrollment is complete for the Phase 3 NEURO-TTRansform trial testing Ionis Pharmaceuticals’ eplontersen in adults with familial amyloid polyneuropathy (FAP). The trial…

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing,…

Challenges with mental health often follow a person being told that they carry a mutation that causes familial amyloid polyneuropathy (FAP), a rare and…

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty…

Novo Nordisk has acquired Prothena’s clinical-stage antibody PRX004, an investigational therapy for people with hereditary transthyretin amyloidosis (ATTR), which also includes familial amyloid…

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy…

Treatment with disease-modifying therapies can extend survival in people with familial amyloid polyneuropathy (FAP), a new study indicates. The study, “Use…

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A…

A single dose of Intellia Therapeutics’ investigational CRISPR/Cas9-based gene-editing therapy NTLA-2001 safely results in a rapid and strong reduction in the levels of…