Rare diseases

News

BioMarin ends year with $4.8bn play for Amicus

BioMarin takes a leap towards its $4bn revenue target with a $4.8bn deal to acquire Amicus, which has two rare disease therapies on the market.

News

Kyverna rises on stiff person syndrome data

Kyverna hopes to file CAR-T therapy miv-cell for approval as the first therapy for stiff person syndrome, a rare and debilitating autoimmune disease.

News

FDA clears first gene therapy for rare genetic disease WAS

Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit.

News

Bayer takes Alport syndrome drug into phase 2

Bayer has started a phase 2a trial of an antibody that it hopes could provide a targeted, potentially disease-modifying therapy for Alport syndrome.

News

Young woman suffering from bleeding disorder, nosebleed

Vaderis drug lends hope to patients with bleeding disease HH...

A trial of Vaderis' AKT inhibitor engasertib finds it could be the first effective treatment for rare disease hereditary haemorrhagic telangiectasia.

News

FDA unveils swift route to market for personalised drugs

FDA reveals a regulatory pathway for personalised therapies based on 'plausible mechanism', drawing on the bespoke gene-editing treatment of Baby KJ.

BioMarin ends year with $4.8bn play for Amicus

Kyverna rises on stiff person syndrome data

FDA clears first gene therapy for rare genetic disease WAS

Bayer takes Alport syndrome drug into phase 2

Vaderis drug lends hope to patients with bleeding disease HH...

FDA unveils swift route to market for personalised drugs

Andexxa reaches the end of the line in the US

How to meet your physicians where they are online

Rare diseases

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