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Ascendis scores FDA approval for weekly dwarfism drug

Children with achondroplasia will soon have the option of a once-weekly - rather than daily - injection, courtesy of Ascendis' newly approved Yuviwel.

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CMS extends deadline for MFN pricing pilot scheme

CMS has extended the deadline for its Medicaid drug pricing pilot, GENEROUS, to "provide interested drug manufacturers more opportunity to join."

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New FDA knockback hits uniQure and Huntington patients hard

uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, delaying the highly anticipated programme.

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CHMP backs Ipsen drug for childhood brain cancer

Ipsen's Ojemda is on course to become the first targeted medicine in the EU for some children with a form of brain cancer known as low-grade glioma.

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NHS "isn't delivering equitable care for rare diseases"

A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.

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