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- PMLiVE

systemic juvenile idiopathic arthritis

- PMLiVE

FDA awards over $38m for advancement of rare disease treatments

Grants will support clinical trials, natural history studies and regulatory science tools

- PMLiVE

Novartis bags CHMP recommendation for prostate cancer drug Pluvicto

The recommendation is for use in combination with androgen deprivation therapy

Patient Centric Approaches for Rare and Ultra-Rare Diseases

Lawrence Bressler, Head of Value & Market Access, Global Rare Diseases at Chiesi, shares how Chiesi is revolutionizing the lives of people living with rare diseases on a global scale....

Impetus Digital

- PMLiVE

Reframing rare to increase earlier diagnosis of Gaucher Disease

Sanofi Genzyme wanted to reach haematologists; the specialist physicians most likely to encounter patients with Gaucher Disease prior to diagnosis. The problem? Gaucher Disease is considered very rare and its...

Create Health

- PMLiVE

Rare diseases: making a difference through education

Medscape Education team up with The Guardian to launch a Rare Disease campaign in the UK.

Medscape Education Global

- PMLiVE

BeiGene’s Brukinsa gets green light from NICE as Waldenström’s macroglobulinemia treatment

The condition occurs in less than 2% of patients with non-Hodgkin lymphomas

- PMLiVE

Novartis announces $300m investment to boost development of next-generation biotherapeutics

The multi-year investment will be implemented across the company’s existing locations in Switzerland, Slovenia and Austria

- PMLiVE

Novartis and BeiGene announce positive phase 3 trial results for hepatocellular carcinoma treatment

Hepatocellular carcinoma accounts for 75-85% of liver cancer diagnoses

- PMLiVE

Novartis and Kaiku Health expand digital health monitoring partnership

The expansion aims to provide improved cancer care to a wider group of patients spanning multiple indications

- PMLiVE

Novartis’ Scemblix receives EC approval for chronic myeloid leukaemia

It is estimated that more than 6,300 people will be diagnosed with CML in Europe every year

- PMLiVE

Novartis to temporarily suspend Huntington’s disease phase 2b study after side effects reported

A data review found that branaplam may cause peripheral neuropathy, which is the result of nerve injury outside the brain and spinal cord

- PMLiVE

Novartis to separate generic drugs division Sandoz to create standalone company

The spin-off will allow shareholders to participate fully in the potential future upside of both companies

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