Rare Disease

Views & Analysis

Unlocking the potential in rare disease research with decent...

A survey by rare disease patient network Raremark found that 86% of the community members asked were interested in taking part in clinical trials.

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Janssen J&J Johnson and Johnson pharmaceutical companies

J&J’s Darzalex Faspro is first US therapy for rare blood dis...

Johnson & Johnson’s Darzalex Faspro has become the first product approved by the FDA to treat light chain (AL) amyloidosis, a rare and often fatal blood cell disorder.

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Young lonely man in a wheelchair on a balcony looking at the nature in the spring

Sarepta helps bankroll new gene therapy startup AavantiBio

Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former employer and three high-profile life sciences

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Chord gets $16m to challenge Alexion with oral NMOSD drug

Swiss biotech Chord Therapeutics has come out of stealth mode with a $16 million first-round financing that will be used to progress a repurposed drug for rare disease neuromyelitis optica

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EMA starts rapid review of Bluebird’s gene therapy for rare ...

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.<

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Novartis' bid to expand use of SMA gene therapy delayed afte...

Analysts have said the FDA’s request for a new trial will delay Novartis’ intrathecal formulation of its spinal muscular atrophy (SMA) gene therapy in older patients until 2023, potentially