Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy…
News
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…
HATTR-PN
NewsEarly 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…
Alnylam Pharmaceuticals’ application for the approval of vutrisiran, its investigational therapy for familial amyloid polyneuropathy (FAP), is being reviewed by the U.S.
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient…
HATTR-PN
NewsAmyl 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…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that…
Enrollment is now completed in the Phase 3 APOLLO-B trial, which is assessing the safety and efficacy of Onpattro (patisiran) to treat heart disease…
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