Breaking news in research

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  • Positive preliminary results from SMA gene therapy trial

    17 April 2019

    AveXis has shared preliminary data from its phase 3 clinical trial (STR1VE) in spinal muscular atrophy type 1. The treatment, Zolgensma, is a gene therapy designed to replace the faulty gene that causes the condition. The trial results so far show that children treated with Zolgensma are living longer and achieving significant development milestones.

    For more information read AveXis’ press release.

  • Wave announces details of its upcoming DMD trial

    16 April 2019

    Today, Wave Life Sciences shared details of its upcoming Phase II/III clinical trial testing suvodirsen in boys with DMD. Suvodirsen is an exon-skipping drug that targets exon 51 within the dystrophin gene. The study, named DYSTANCE 51, will start in July 2019 and enrol boys who are between 5 and 12 years of age. Participants will receive suvodirsen or placebo once a week for 48 weeks.

    For further information, please read Wave’s press release.

  • Audentes starts therapy development for DMD and myotonic dystrophy

    8 April 2019

    Today, Audentes Therapeutics has announced that it will begin the development of new genetic medicines for Duchenne muscular dystrophy and myotonic dystrophy type 1. These therapies will aim to address the underlying causes of these conditions by delivering genetic information into the body. Audentes currently has an AAV-based gene therapy for myotubular myopathy in early-stage clinical trial.

    For more information read Audentes’ community letter and press release.

  • Sarepta announces positive preliminary results from DMD trial

    29 March 2019

    Sarepta Therapeutics has released positive preliminary results from one of its clinical trials testing exon skipping in Duchenne muscular dystrophy. The study drug, casimersen, targets exon 45 of the dystrophin gene. The investigators have found increased levels of dystrophin in muscles of boys treated with the drug for 11 months. Information on the safety and efficacy of the treatment will only be available at a later stage. However, based on these initial results, Sarepta hopes to request approval from the US Food and Drug Administration in the middle of 2019.

    For more information, read Sarepta’s press release.

  • Santhera prepares to request approval for idebenone in DMD

    28 March 2019

    Santhera Pharmaceuticals has provided an update on its plans to request approval for Puldysa in Duchenne muscular dystrophy. The active ingredient in this drug is idebenone, an antioxidant that has the potential to slow down respiratory decline in people with the condition. Back in 2017, Santhera requested authorisation for idebenone under the brand name Raxone, but the application was refused by the European Medicines Agency (EMA). Since then, the company has gathered additional data on the drug’s efficacy and changed the brand name in order to fulfil requests from regulators. Santhera is now planning to send a new application to EMA in the second quarter of 2019.

    For more information, read Santhera’s press release.

  • Update on Sarepta’s DMD gene therapy trial

    26 March 2019

    Sarepta Therapeutics has provided an update on the four boys participating in its gene therapy trial for Duchenne muscular dystrophy. It’s now been nine months since the boys were treated. They are all doing well and no serious safety concerns have been reported. Tests have also shown that their creatine kinase levels have decreased and their muscle function appears to be better than what is usually seen in Duchenne patients of the same age. Although the boys will continue to be followed up for 36 months in total, these results are promising and the company is now recruiting for a Phase II trial in USA.

    For more information, access the conference call slides and audio here.

  • Polaris DMD trial starts recruiting in the UK

    25 March 2019

    We have learnt today from Catabasis that its Polaris DMD trial has started enrolling patients at Bristol Royal Hospital for Children. This is a global phase 3 trial testing edasalonexent (CAT-1004) in boys with Duchenne muscular dystrophy. There are three other UK sites planning to start recruitment soon: Evelina London Children’s Hospital, Great Ormond Street Hospital and Royal Manchester Children’s Hospital. All interested families are urged to contact

    Read more about the Polaris DMD trial here.

  • Update from FSHD drug screening programme

    21 March 2019

    Facio Therapies announced today that it has completed a comprehensive search for new FSHD drug candidates. The company has identified 100+ compounds that show some level of activity in lab tests. The next step will be to prioritise the most promising compounds to investigate in more detail.

    For more information, read Facio’s press release.

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