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

Genetic testing hopes to speed up drug development in rare e...

When it comes to fighting rare diseases, the biggest barrier to overcome is often the lack of knowledge around the condition.