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Samsung Bioepis

- 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

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

FDA unveils new voucher programme to accelerate drug review process

The programme is designed to reduce the review time for companies supporting US national interests

- 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

- PMLiVE

Novartis’ Fabhalta granted FDA approval to treat ultra-rare kidney disease C3G

Approximately half of C3G patients progress to kidney failure within ten years of diagnosis

- PMLiVE

Novartis shares positive phase 3 results for spinal muscular atrophy gene therapy

The company said it is planning to file regulatory applications for OAV101 IT this year

- PMLiVE

NICE recommends Pharming’s Joenja as first treatment for ultra-rare immune disease APDS

Approximately 40 to 50 people in the UK are known to be living with the inherited disorder

- PMLiVE

Amgen’s Uplizna shows promise in phase 3 generalised myasthenia gravis study

Regulatory submissions are expected to be complete in the first half of this year

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