Rare Disease

News

Second death in gene therapy trial prompts investigation

A second death in a study of Novartis’ spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) – a potential blockbuster for this rare disease – has prompte

News

Ipsen launches new syringe for rare disease blockbuster

Ipsen will be launching a new pre-filled syringe for its blockbuster rare disease drug Somatuline (lanreotide) in the EU.

News

Bernie Sanders has helped create momentum behind the 'Yes On 61' campaign

Sanders calls on FDA to allow low-cost rivals to $375k Catal...

US Senator Bernie Sanders says the FDA should allow pharmacies and generic manufacturers to make low-cost copies of Catalyst Pharma’s rare disease drug Firdapse, which is at the heart of a

Views & Analysis

engineering and genetic editing through the Crispr technique. DNA chain breaking down. 3D rendering

Gene addition – a new chapter in treating rare diseases

The scientific advancements of the past 30 years have transformed our understanding of the potential of gene therapy and how it may be used to tackle rare diseases.

Views & Analysis