Breaking news in research

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  • Acceleron discontinues drug development for CMT

    10 March 2020

    Today, Acceleron announced topline results from its Phase II ACE-083 trial for Charcot-Marie-Tooth Disease. Although the drug increased the size of the muscles it was injected into, this did not translate into a clinical benefit i.e. there was no improvement in muscle strength or function. Unfortunately, this means that Acceleron is discontinuing development of ACE-083 for CMT.

    ACE-083 is a drug that inhibits a family of proteins that negatively regulate muscle growth (including myostatin).

    For more information, read Acceleron’s press release.

  • First CNM patient receives anti-sense drug

    5 March 2020

    Dynacure have announced that the first person in its Phase I/II trial, Unite-CNM, has received the drug DYN101. This is the first time that anyone with centronuclear myopathy (CNM) has received an anti-sense drug.

    DYN101 is an antisense drug designed to switch off DNM2, a gene that is overactive in CNM.

    For more information, read Dynacure’s press release.

  • More positive news from SMA SUNFISH trail

    6 February 2020

    PTC Therapeutics have shared the clinical data presented at the International SMA Europe Conference in France.

    The study showed improvement in muscle function in people with SMA type 2 and 3 when treated with risdiplam over a period of 12 months. Children aged 2-5 years, showed improvement compared to those not receiving the drug. In people older than 5, the progression of the condition stabilised.

    The company will be presenting the data in a conference call for investors at 1pm today. It can be accessed by dialing (973) 935-8152 five minutes before the start of the call and entering the passcode 7757508.

    For more information, read PTC’s press release.

  • FSHD drug granted orphan drug status by FDA

    29 January 2020

    Fulcrum Therapeutics has announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) to losmapimod for the treatment of patients with FSHD. This designation gives Fulcrum certain financial benefits that will help to lower the cost of developing the drug. Losmapimod has been shown to “switch off” DUX4 in cells originating from people with FSHD. The safety and efficacy of the drug is currently being tested in a Phase 2 clinical trial. The results from this trial are expected later in 2020. For more information, please read Fulcrum’s press release.

  • Duchenne trial to extend to non-ambulatory boys and men

    8 January 2020

    Pharmaceutical company, Catabasis, and charity Duchenne UK have announced a partnership to study the drug, edasalonexent, in the non-ambulatory DMD population.

    Edasalonexent works by turning off an enzyme called NF-kB, which is known to be overactive in DMD. It has been shown to slow the progression of Duchenne and is currently being evaluated in a phase 3 trial in boys aged four to seven.

    The new study will evaluate the safety and efficacy of the drug in non-ambulatory boys and men and will be recruiting in the UK.

    Read the press release from Catabasis here.

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