Surviving as a rare disease biotech during COVID-19

With trial delays, social distancing, and falls in charitable funding, the coronavirus pandemic presents some serious challenges for nonprofit orphan disease biotechs like Cure Rare Disease

The ALS patient journey

Newron's shares dive after rare disease drug failure

Newron’s share price has collapsed after the biotech axed development of its drug sarizotan for the rare genetic brain disorder Rett Syndrome following the failure of a key trial. 

FDA delays decision on Roche's SMA drug to review extra data

The FDA has extended its review of Roche’s spinal muscular atrophy (SMA) drug risdiplam after the pharma submitted additional data.

Chiesi ups its focus on rare diseases

Italy’s Chiesi has created a new business unit aimed at increasing its presence in the market for rare disease drugs.

Novartis publishes long-term data from SMA gene therapy

Novartis has produced data demonstrating its Zolgensma gene therapy produces long term benefits in the rare muscle wasting disease spinal muscular atrophy – information that will be closely