News

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

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 pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

Novo Nordisk Acquires Prothena’s PRX004, Plus Full ATTR Program

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 polyneuropathy (FAP), as well as other non-hereditary forms of the disease. While the company initially will focus on developing PRX004 for the treatment of ATTR…

Nominations Open for 2022 Eurordis Black Pearl Awards

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

Early Data Favorable for NTLA-2001 Gene-editing Therapy

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 the damaging transthyretin (TTR) protein, according to interim data from the first six familial amyloid polyneuropathy (FAP) patients given the therapy in a Phase…

FDA to Decide on Vutrisiran’s Possible Approval by April 2022

Alnylam Pharmaceuticals’ application for the approval of vutrisiran, its investigational therapy for familial amyloid polyneuropathy (FAP), is being reviewed by the U.S. Food and Drug Administration (FDA), with a decision expected by April 14 of next year. The agency has indicated that it is not currently planning…

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

Amyl Raises Funds to Develop Therapies for Amyloidosis

Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance therapeutic candidates for familial amyloid polyneuropathy (FAP) and other forms of amyloidosis. “The ClariTY platform offers the unique opportunity to generate therapeutic candidates able to target all amyloid fibril…