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rare diseases

- PMLiVE

UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

- PMLiVE

Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

- PMLiVE

UCB releases new data on Bimzelx for hidradenitis suppurativa

The disease affects around 1% of the population in most studied countries

- PMLiVE

Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

- PMLiVE

AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

- PMLiVE

AstraZeneca reports positive results from rare disease trial

HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

- PMLiVE

UCB’s Fintepla shows positives results for Lennox-Gastaut syndrome

The study demonstrated a sustained reduction in seizures for patients

- PMLiVE

UCB shares promising results for fenfluramine in ultra-rare form of epilepsy

CDKL5 deficiency disorder occurs in approximately one in 40,000 to 60,000 live births

- PMLiVE

UCB’s Bimzelx demonstrates lasting efficacy in phase 3 psoriatic arthritis trials

Approximately 125 million people worldwide are affected by some form of psoriasis

- PMLiVE

Amgen’s Uplizna approved by FDA as first treatment for rare disease IgG4-RD

Immunoglobulin G4-related disease affects approximately 20,000 people in the US

- PMLiVE

FDA approves Soleno’s Vykat XR as first treatment for hyperphagia in Prader-Willi syndrome

The rare genetic disorder affects an estimated one in every 15,000 newborns in the US

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