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    • aromatic L-amino acid decarboxylase deficiency

    - PMLiVE

    FDA approves PTC Therapeutics’ Sephience to treat rare metabolic disorder PKU

    Phenylketonuria affects one in every 13,500 to 19,000 newborns in the US

    - PMLiVE

    J&J’s Darzalex recommended by CHMP to treat smouldering multiple myeloma

    If approved, Darzalex would be the first therapy approved in the EU for SMM

    - PMLiVE

    Organon/Henlius announce EMA validation for Perjeta biosimilar HLX11

    The reference medicine holds approvals for multiple HER2-positive breast cancer indications

    - PMLiVE

    Rare diseases: Novartis’ Fabhalta recommended by CHMP for C3 glomerulopathy

    Approximately one to two people per million worldwide are diagnosed with the ultra-rare kidney disease every year

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    Rare diseases: CHMP recommends Krystal’s Vyjuvek for dystrophic epidermolysis bullosa

    There is currently only one treatment authorised for the ultra-rare genetic blistering disease

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    CHMP recommends Bavarian Nordic’s chikungunya vaccine for individuals aged from 12 years

    Approximately 480,000 cases of the mosquito-borne viral disease were reported globally in 2024

    - PMLiVE

    Gilead’s seladelpar recommended by CHMP to treat primary biliary cholangitis

    The rare liver disease affects approximately 15 per 100,000 people in Europe

    - PMLiVE

    GSK’s Jemperli receives positive CHMP opinion for expanded endometrial cancer use

    Approximately 121,000 people are diagnosed with primary advanced or recurrent endometrial cancer each year in Europe

    - PMLiVE

    Novartis gains rights to PTC’s Huntington’s programme in deal worth up to $2.9bn

    The genetic disorder affects one in every 10,000 to 20,000 people in the US

    - PMLiVE

    MHRA urges companies to prepare for Windsor Framework for medicines

    The new UK-wide arrangements for medicines will come into effect on 1 January 2025

    Biogen Idec building

    Biogen/Eisai’s Alzheimer’s drug lecanemab receives CHMP recommendation

    The neurodegenerative disorder affects an estimated 6.9 million people in Europe

    - PMLiVE

    Novartis’ Kisqali receives CHMP recommendation for early breast cancer patients

    Approximately 70% of all breast cancer patients have HR-positive/HER2-negative disease

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