Rare diseases

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Janssen J&J Johnson and Johnson pharmaceutical companies

Janssen files Darzalex Faspro for rare disease light chain a...

Janssen has filed a new formulation of its Darzalex in the US for the rare and potentially fatal disease light chain (AL) amyloidosis.

Views & Analysis

Searching for answers in rare epilepsy

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases.

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BioMarin files achondroplasia drug after rejection for haemo...

US biotech BioMarin has hit back with a filing for a rare disease drug, after suffering what was described by analysts as a “major setback” when the FDA rejected its gene therapy for haemop

News

Healx uses AI to find drugs for Angelman Syndrome

The UK-based AI technology company Healx has begun a partnership with the Foundation for Angelman Syndrome Therapeutics (FAST), focusing on developing new therapies for the rare neurogeneti

News

NS Pharma takes on Sarepta as FDA approves DMD drug

The FDA has approved a new therapy for the rare muscle wasting disease Duchenne muscular dystrophy (DMD) as Japan’s NS Pharma takes on Sarepta and its controversially approved rival.

Views & Analysis