Breaking news in research

The latest news related to research, industry and clinical trials. You can also visit our Research News section for more detailed research stories.

  • Sarepta launch Managed Access Programme for Exondys51

    19 July 2017

    Sarepta Therapeutics today announced that it has initiated a Managed Access Programme for Exondys51. Managed access programmes provide a mechanism through which physicians can legally and ethically prescribe an unapproved therapy to patients who meet pre-specified medical criteria and can secure funding. According to Sarepta’s website, the programme is being offered in the UK and physicians have to make requests on behalf of their patients. We will keep you updated.

    For more information, read Sarepta’s press release.

  • Duchenne cardiac cell therapy receives Rare Paediatric Disease Designation

    19 July 2017

    Capricor Therapeutics has announced that the US Food and Drug Administration (FDA) has granted Rare Paediatric Disease Designation to CAP-1002 for the treatment of Duchenne muscular dystrophy. Under the FDA Rare Pediatric Disease Priority Review Voucher Program, Capricor could receive certain benefits if CAP-1002 is approved.

    CAP-1002 is a cardiac cell therapy that is currently being tested in a phase 1/2 trial called HOPE. Results at the six month mark showed that participants given CAP-1002 had significant improvements in heart and arm function. The treatment was also well-tolerated and safe. Capricor expects to report 12-month results from the HOPE trial in the fourth quarter of 2017.

    For more information, read Capricor’s press release.

  • Sarepta and BioMarin resolve patent dispute

    18 July 2017

    Sarepta Therapeutics and BioMarin Pharmceuticals today announced that they have resolved a legal patent dispute over Sarepta’s sale of Exondys51 and future exon skipping products. This is good news for the Duchenne community as the dispute could have potentially created problems with accessing the drugs.

    For more information, read this press release.

  • CRISPR gene editing reverses paralysis in mice with congenital muscular dystrophy

    18 July 2017

    Scientists from the Hospital of Sick Children in Toronto have shown that gene-editing tool CRISPR can correct the underlying genetic mutation in mouse models with merosin-deficient congenital muscular dystrophy (MDC1A). This led to an improvement in muscle strength and removed all signs of paralysis in the mice. Principal investigator of this study is Dr Ronald Cohn, who presented some of these results at our patient information day held in London in March. The full study has now been published in the prestigious medical journal, Nature Medicine.

    For more information, watch this video of Dr Cohn and read this press release.

  • Mallinckrodt drug receives Orphan Drug Designation for the treatment of Duchenne

    13 July 2017

    Mallinckrodt Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to MNK-1411 for the treatment of Duchenne muscular dystrophy. This special status gives companies certain incentives that help to speed up the development of their rare disease products.

    For more information, read Mallinckrodt’s press release

  • Sarepta and Genethon form partnership

    21 June 2017

    Sarepta Therapeutics announced today that it is partnering with Genethon, a non-profit organisation that develops gene therapies for rare diseases. Genethon is currently developing a micro-dystrophin gene therapy, which could be a potential treatment for Duchenne muscular dystrophy. This partnership will help to advance this research by bringing together the expertise of Sarepta and Genethon.

    For more information, read Sarepta’s press release.

    Update 30/6/17 – The Sarepta-Genethon partnership has positive implications for the UNITE-DMD project that we are co-funding with Action Duchenne and the French Muscular Dystrophy Association (AFM). Read our full statement here.

  • Facio raise €4.8mil for FSHD drug screening

    20 June 2017

    Facio Therapies announced today that it has raised €4.8 million from a group of shareholders, which includes families affected by FSHD, the French charity Amis FSH and drug discovery partner, Evotec. Facio and Evotec have been screening hundreds of drugs for their ability to block the production of DUX4 protein in muscle cells with FSHD. The €4.8mil funding will help to accelerate this screening and the identification of pre-clinical candidates.

    For more information, read Facio’s press release.

  • Former cancer drug could be beneficial for Duchenne

    14 June 2017

    A new study has shown that a drug originally developed to target cancer could be a potential treatment for Duchenne muscular dystrophy. The researchers screened 350,000 drugs and found that one called SU9516 helped to improve the muscle function of cell and mouse models with Duchenne. They now plan to work with medicinal chemists to improve the specificity of SU9516 and to remove its toxic anti-cancer components. This will hopefully lead to a safer version of the drug that could potentially be tested in people with Duchenne muscular dystrophy.

    For more information, read the NIH press release.

  • FDA to meet with PTC about Translarna

    8 June 2017

    PTC Therapeutics has announced that it is scheduled to meet with the FDA Peripheral and Central Nervous Systems Drugs Advisory Committee on 28 September 2017. During this meeting, the company’s licensing application for Translarna will be reviewed. For more information, read PTC’s press release and this news story.

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