Pharmafile Logo
  • - PMLiVE
  • - PMLiVE
  • Diversity, Equity and Inclusion
  • Communiqué 53 Cover Image

    • Sarepta

    - PMLiVE

    Lilly and MeiraGTx partner in $475m ophthalmology deal

    The companies will develop and commercialise therapies for genetic conditions

    The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter

    By Sarah Mohamad and Lauren Coleman-Bennett

    Amiculum

    What do people look for in gene therapy materials? Results from our survey of patients and relatives

    By Sam Fraser

    Amiculum

    - PMLiVE

    CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix

    The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US

    - PMLiVE

    NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease

    About 15,000 people in England are living with the inherited blood disorder

    - PMLiVE

    Roche/Sarepta announce two-year results for Duchenne muscular dystrophy gene therapy

    The muscle-wasting disorder is estimated to affect one in every 5,000 male births worldwide

    - PMLiVE

    MHRA approves Italfarmaco’s Duvyzat to treat Duchenne muscular dystrophy

    The severe neuromuscular disease affects approximately 2,500 people in the UK

    - PMLiVE

    NICE recommends Santhera’s Duchenne muscular dystrophy drug Agamree

    Approximately 1,700 patients with the muscle-wasting disorder in England are expected to benefit from the decision

    - PMLiVE

    Vertex and Orum partner on gene editing conditioning drugs in deal worth $945m

    The agreement gives Vertex the rights to use Orum’s targeted protein degradation technology

    - PMLiVE

    FDA expands indication for Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys

    The neuromuscular condition is estimated to affect one in every 3,500 male births worldwide

    - PMLiVE

    FDA approves Italfarmaco’s Duvyzat as first nonsteroidal Duchenne muscular dystrophy drug

    The neuromuscular genetic disease is estimated to affect one in every 3,500 to 6,000 male births worldwide

    - PMLiVE

    Santhera’s Duchenne muscular dystrophy drug shows promise as long-term treatment

    The muscle-wasting disorder is estimated to affect one in every 3,500 male births worldwide

    Subscribe to our email news alerts

    Latest content

    Latest intelligence

    Quick links