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- PMLiVE

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Medscape Education: Rare Diseases Fibrodysplasia Ossificans Progressiva

Rare Disease Day is on February 28.Listen to Nicky explain her daughter's journey with fibrodysplasia ossificans progressiva.Many patients face long waits for accurate diagnosis and treatment, making it critically important...

Medscape Education Global

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NICE publishes final draft guidance recommending three COVID-19 treatments

The medicines have shown promise in treating COVID-19 and been found cost-effective

- PMLiVE

SMC enables access to Kyowa Kirin’s Crysvita for adults with rare genetic disease

XLH is a life-long genetic disease that causes abnormalities in the bones, muscles and joints

- PMLiVE

Sanofi’s enzyme replacement therapy available on NHS to Pompe disease patients

The rare genetic disorder affects around one in every 40,000 people in the UK

- PMLiVE

Roche’s Polivy recommended by NICE for lymphoma therapy

Around 5,500 people in England are diagnosed with the aggressive blood cancer each year

- PMLiVE

Gilead’s CAR-T cell therapy recommended by NICE for lymphoma

Yescarta is the first CAR T-cell therapy recommended for routine use on the NHS

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Alnylam’s RNAi therapeutic recommended by NICE for hereditary form of amyloidosis

Amvuttra is given every three months, compared to every few weeks with existing therapies

- PMLiVE

Takeda’s all-oral multiple myeloma triplet therapy recommended by NICE

Ninlaro with lenalidomide and dexamethasone improved clinical outcomes

- PMLiVE

NICE recommends wider use of statins to cut risk of heart attack and stroke

The guidance comes after new evidence was considered on the side effects and safety of statins

- PMLiVE

Ipsen to acquire rare disease specialist Albireo in deal worth over $950m

The deal will strengthen the biopharma’s liver disease pipeline with the addition of Bylvay

- PMLiVE

Amgen announces plans to acquire Horizon for $27.8bn in biggest pharma deal for 2022

The acquisition will see Amgen pay $116.50 for each Horizon share in cash

- PMLiVE

ICR calls for further discussions following NICE’s rejection of breast cancer drug

Olaparib has been shown to cut the risk of cancer recurrence and improve survival

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