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.

  • Acceleron update on myostatin inhibitors

    20 September 2017

    Acceleron Pharma has released an update on its neuromuscular programme. It is currently developing two drugs that aim to increase muscle mass by blocking myostatin. ACE-083 is currently being tested in people with facioscapulohumeral dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) in phase 2 clinical trials. Preliminary results from the FSHD trial are expected in late 2017. ACE-2494, which works in a similar way to ACE-083 but is administered systemically, has shown promising results in preclinical studies.  A phase 1 study testing ACE-2494 is expected to be initiated later this year.        

    For more information, please read Acceleron Pharma’s press release.

  • Update on Roche’s SUNFISH study

    18 September 2017

    SUNFISH is a two-part clinical study investigating the drug, RG7916, in people with type 2 or 3 SMA.  Part 1 assessed the safety and tolerability of different doses of RG7916. This is now complete and an appropriate dose has been chosen. The safety and efficacy of this dose will now be assessed in part 2 of the study, which will begin recruiting soon.

    Please read Roche’s community letter for more information.

  • Update from Alexion Pharmaceuticals’ extension study

    14 September 2017

    Alexion Pharmaceuticals has released interim results from their extension trial of the original REGAIN phase 3 study. This study is testing eculizumab in individuals with refractory generalised myasthenia gravis, a rare form of myasthenia gravis that does not respond to conventional therapies.So far results have shown sustained clinical improvement in participants who took eculizumab throughout the extension study. This study is ongoing and planned to continue until January 2019.

    For more information please read Alexion Pharmaceuticals’ press release.

  • Positive results from SKIP-NMD trial

    06 September 2017

    Sarepta Therapeutics has announced the latest results from the 4053-101 study (see SKIP-NMD project for more details). This phase 1/2 clinical study is assessing the safety and efficacy of golodirsen, a drug that skips exon 53 in individuals with Duchenne muscular dystrophy. Muscle biopsies from the treated participants showed that the drug was able to skip exon 53. This had a positive effect on dystrophin production.

    Please read Sarepta Therapeutics’ press release for more information.

  • Update on the development of Raxone

    05 September 2017

    Santhera Pharmaceuticals has given an update on the development progress of Duchenne drug, Raxone. EMA’s Committee for Medicinal Products for Human Use (CHMP) is currently reviewing Raxone as a potential treatment for patients with respiratory decline who are not taking steroids. The CHMP is expected to give an opinion shortly. The drug is currently available to patients who fit certain criteria under the UK’s Early Access to Medicines Scheme (EAMS) (read this news story for more information). Santhera is also assessing the safety and efficacy of Raxone in patients receiving steroids in a phase 3 trial called SIDEROS.

    For more information, please read Santhera Pharmaceuticals’ press release.

  • Ultragenyx stops Ace-ER development for GNE myopathy

    23 August 2017

    Ultragenyx Pharmaceuticals has announced results from its phase 3 clinical trial testing aceneuramic acid extended release (Ace-ER) in adults with GNE myopathy. Although Ace-ER was well tolerated, it did not significantly improve muscle strength. Unfortunately this means that Ultragenyx is discontinuing clinical development of Ace-ER. However, Ultragenyx are planning to release data that could be valuable for development of future therapies.

    For more information, please read Ultragenyx’s press release and community email

  • Update on gene therapy for myotubular myopathy

    10 August 2017

    Biotechnology company Audentes Therapeutics has announced that it plans to initiate a phase 1/2 trial for boys with X-linked myotubular myopathy (X-MTM) in the third quarter of 2017. The trial is called ASPIRO and will test the safety and preliminary efficacy of a gene therapy called AT132. AT132 uses an adeno-associated virus (AAV) to deliver a healthy copy of the MTM gene into the body.

    For more information, read Audentes’ press release and these FAQs.

  • Mitochondrial Duchenne drug enters clinical development

    8 August 2017

    Biotechnology company MitoBridge today announced that is initiating clinical testing for MA-0211, a drug that modulates mitochondrial function and could be a potential treatment for Duchenne muscular dystrophy. The safety of MA-0211 will be evaluated in healthy volunteers in a phase 1 clinical trial, which will then provide the basis for a clinical programme for people with Duchenne.

    For more information, visit the MitoBridge website or watch this video presentation.

  • Gene therapy shows promise in severe DMD mouse model

    28 July 2017

    Researchers from the University of Missouri and Solid Biosciences have shown that their investigational micro-dystrophin gene therapy improved muscle function of a severe mouse model of Duchenne muscular dystrophy. Read this press release for more information.

  • Capricor report on FDA meeting

    28 July 2017

    Capricor Therapeutics has released notes from its recent meeting with the US Food and Drug Administration (FDA). These indicate that the FDA is supportive of Capricor’s proposed development plan for CAP-1002, an investigational cardiac cell therapy for Duchenne muscular dystrophy. Capricor plans to use the Performance of the Upper Limb (PUL) as the primary efficacy endpoint for upcoming clinical trials. PUL is an outcome measure designed to assess upper limb function in ambulant and non-ambulant patients with Duchenne.

    For more information, read Capricor’s press release.

  • Sarepta launch Managed Access Programme for Exondys 51

    19 July 2017

    Sarepta Therapeutics today announced that it has initiated a Managed Access Programme for Exondys 51. 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.

    UPDATE 21 JULY – Read our news story for the latest on the Exondys 51 Managed Access Programme

  • 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

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