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- PMLiVE

fast track appraisal

- PMLiVE

Ipsen’s cabozantinib receives NICE approval for advanced hepatocellular carcinoma

The treatment showed a significant improvement in overall survival versus placebo

- PMLiVE

Merck’s Keytruda receives NICE recommendation as breast cancer treatment option

Patients will now be able to access immunotherapy at an earlier stage in their treatment journey

- PMLiVE

Alexion’s Ultomiris shows promise in neuromyelitis optica spectrum disorder phase 3 trial

Ultomiris-treated patients showed zero relapses with a median treatment duration of 73 weeks

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NICE recommends Grifols’ Tavlesse for chronic immune thrombocytopenia

Patients with chronic ITP may live with an increased risk of severe bleeding events

- PMLiVE

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

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

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

Bristol Myers Squibb’s active ulcerative colitis treatment bags NICE recommendation

Phase 3 trial results of Zeposia showed statistically significant results in response and remission

- 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

Merck bags NICE recommendation for renal cell carcinoma adjuvant treatment

Patients will now be able to access immunotherapy at an earlier stage in their treatment pathway

- 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

NICE rejects AstraZeneca and MSD’s Lynparza as NHS prostate cancer treatment

The drug will not be available as a treatment option for men with prostate cancer in England and Wales

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