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rare diseases

This page shows the latest rare diseases news and features for those working in and with pharma, biotech and healthcare.

FDA rejects Sarepta’s new Duchenne drug over safety fears

FDA rejects Sarepta’s new Duchenne drug over safety fears

The company is also investing in gene therapies for DMD and a range of other rare diseases as well as multiple sclerosis.

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Approximately 120 fully matching, plus 245 partially matching documents found.

Latest Intelligence

  • Real-world studies Real-world studies

    myriad of information, from safety monitoring and drug utilisation studies of marketed products in the EU to routine safety monitoring of marketed products and filling evidentiary gaps for particular rare diseases

  • The Cystic Fibrosis Buyers’ Club The Cystic Fibrosis Buyers’ Club

    HIV/AIDS and radical patient activists. The most energised and even radical patient activism has tended to occur in rare, life- threatening diseases – the most influential being the HIV/AIDS movement

  • Streamlining clinical trials – why better data use is key Streamlining clinical trials – why better data use is key

    treatments for rare diseases, clinical trial speed and efficiency need to be addressed”.

  • 25 Women Leaders in UK Healthcare 25 Women Leaders in UK Healthcare

    4. JAYNE SPINK . Rare disease patient champion. Medical research is expanding our understanding of genetics and genetically inherited rare diseases daily, but this progress is agonisingly slow if you are living ... Genetic Alliance UK is home to Rare

  • 25 Women Leaders in UK Healthcare (part 2) 25 Women Leaders in UK Healthcare (part 2)

    Prior to joining Gyroscope, Soraya served as SVP Chief Medical Officer of Lysogene, a clinical stage biotech developing gene therapy products in rare central nervous system diseases, serving in global gene ... Dr Larissa Kerecuk is a doctor who has made

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Approximately 16 fully matching, plus 83 partially matching documents found.

Latest appointments

  • New faces at imc, GCI Health and Lighthouse US New faces at imc, GCI Health and Lighthouse US

    Additionally, she has also served as a member of the board of management of the ABPI, where she championed access and funding for patients with rare diseases.

  • New faces at Lucid, 90Ten and Page & Page New faces at Lucid, 90Ten and Page & Page

    At Synergy, Bates was project director, working in rare diseases. She was also a publisher at Wounds UK and Wounds international, heading up the medical education division. ... Elaine Downey joins Vivid – Lucid’s rare and specialist diseases agency

  • Weekly industry appointments Weekly industry appointments

    Appleby, who will succeed CGT Catapult’s first CSO Johan Hyllner, currently serves as chief scientific officer for cell and gene therapy at GlaxoSmithKline’s (GSK) rare diseases unit.

  • Paris biocluster appoints Stéphane Roques as new chief Paris biocluster appoints Stéphane Roques as new chief

    He has also chaired the start-up Genosafe and launched the Institute of Biotherapies for Rare Diseases. ... He also chaired the start-up Genosafe and launched the Institute of Biotherapies for Rare Diseases.

  • Gamida Cell strengthens leadership team Gamida Cell strengthens leadership team

    Their combined experience in corporate strategy, business development and commercialisation will be invaluable to the company’s continued growth as we advance our clinical programmes in blood cancers and rare genetic ... diseases.”.

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Approximately 1 fully matching, plus 53 partially matching documents found.

Latest from PMHub

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Approximately 14 fully matching, plus 58 partially matching documents found.

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