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    • Translarna

    - 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

    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

    Positive CHMP opinion issued for PTC Therapeutics’ Upstaza

    The treatment for AADC will be the first marketed gene therapy to be administered directly into the brain

    - PMLiVE

    FDA rejects Sarepta’s new Duchenne drug over safety fears

    FDA gave no warnings of concerns, says chief exec

    - PMLiVE

    Duchenne UK backs Evox dystrophin delivery project

    Funding will drive preclinical studies on dystrophin-bearing exosomes

    - PMLiVE

    Sarepta soars on positive Duchenne data

    Trial results position Sarepta as a “leader in the field” according to analysts

    - PMLiVE

    FDA reviewer unimpressed with PTC’s Translarna

    Finds the muscular dystrophy treatment's evidence to be 'not persuasive'

    - PMLiVE

    Pfizer buys gene therapy specialist Bamboo

    Expands pipeline with Duchenne muscular dystrophy candidate in deal worth $700m

    National Institute for Health and Care Excellence NICE logo

    NICE backs PTC’s muscular dystrophy drug Translarna

    Drug to be available on the NHS for DMD patients aged five and over

    - PMLiVE

    Santhera facing long delay for muscular dystrophy drug in US

    FDA rejects accelerated marketing application for Raxone to await phase II trial results

    Biomarin

    BioMarin gives up on muscular dystrophy drug

    Makes ‘difficult but necessary’ decision to abandon exon-skipping therapy Kyndrisa

    National Institute for Health and Care Excellence NICE logo

    NICE backs first Duchenne muscular dystrophy drug

    Reverses original decision to reject the therapy in favour of more data

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