Novartis offers reimbursement options as EU backs costly SMA...

Novartis’ AveXis unit has said European regulators have approved its Zolgensma (onasemnogene abeparvovec) gene therapy for the rare childhood wasting disease spinal muscular atrophy (SMA),

Takeda posts surprise profit a year after Shire mega-merger

Japan’s Takeda has posted a surprise profit after its $59 billion merger with Shire just over a year ago, as the company shakes off the huge debts it incurred to get the deal done. 

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.