Rare diseases

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.

Patients

Why rare kidney diseases must be part of the CKD conversatio...

A look at the benefits of genetic testing in diagnosis of rare kidney diseases, from HealthLumen.

News

Dismay as FDA declines to approve Biohaven's ataxia drug

Biohaven's troriluzole candidate for spinocerebellar ataxia (SCA) has been turned down by the FDA, in another sign of a tougher stance by the agency.

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

Why rare kidney diseases must be part of the CKD conversatio...

Dismay as FDA declines to approve Biohaven's ataxia drug

CHMP decision levels the playing field for GSK's RSV jab

How to meet your physicians where they are online

Rare diseases

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