Rare diseases

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Multicoloured letters with AUTISM spelled in the middle

FDA approves leucovorin – but not for autism

The FDA stops short of approving leucovorin for autism, but will its clearance for ultra-rare condition CFD-FOLR1 lead to more off-label prescribing?

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Alfasigma licenses liver drug from GSK in $690m deal

With an FDA verdict looming, GSK has signed an eleventh-hour deal to give Alfasigma rights to its primary biliary cholangitis therapy linerixibat.

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PepGen slumps as FDA places hold on lead drug

Shares in PepGen fell sharply after the FDA placed a partial clinical hold on its lead drug for rare neuromuscular disease DM1.

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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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New biotech Atrium born out of Novartis/Avidity marriage

Atrium Therapeutics has launched with $270m in funding and two preclinical siRNA candidates for rare genetic cardiomyopathies.

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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.

FDA approves leucovorin – but not for autism

Alfasigma licenses liver drug from GSK in $690m deal

PepGen slumps as FDA places hold on lead drug

Ascendis scores FDA approval for weekly dwarfism drug

New biotech Atrium born out of Novartis/Avidity marriage

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

Remepy's hybrid Parkinson's therapy poised for phase 3

Discover a new era of healthcare panel engagement: Grounded ...

Rare diseases

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