New drugs approved by the FDA are prolonging the lives of those affected.

Alnylam Canada ULC is pleased to announce it has received a positive recommendation from Canada's Drug Agency (CDA) for the public reimbursement of its RNAi therapeutic, AMVUTTRA®.1 In December 2025, ...

An amyloidosis diagnosis can take upwards of 2 years.Initial symptoms often are nondescript, and irreversible organ damage frequently occurs before disease confirmation.

A research team at Kumamoto University has discovered that a natural compound found in pomegranate leaves and branches can ...

Intellia said the FDA removed the clinical hold on application for the Phase 3 clinical trial of nexiguran ziclumeran. The study is a randomized, double-blind, placebo-controlled trial evaluating the ...

Prothena Corporation plc kept at Hold after mixed phase 2 results; key phase 3 readouts expected 2029. Click for this updated ...

Intellia Therapeutics Inc. NTLA shares are up on Monday following the FDA’s decision to lift the clinical hold on its ...

Thank you, Jason. After that, we'll provide updates on the significant progress we've made with lonvo-z, which is being ...

By Christy Santhosh March 2 (Reuters) - Intellia Therapeutics said on Monday the U.S. Food and Drug Administration has lifted ...

Networked models of care have the potential to provide better access to specialist care for rare diseases in the NHS. The government committed to establishing an innovative networked model of care for ...

Researchers have identified a natural compound from pomegranate leaves and branches that can directly dismantle the protein aggregates responsible for transthyretin amyloidosis.

We are very pleased to have aligned with the FDA on the path forward for our MAGNITUDE clinical trial, with measures designed ...