Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice

Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice: "Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscle tissue, according to a paper published in The Journal of Cell Biology. The study, "Osteopontin ablation ameliorates muscular dystrophy by shifting macrophages to a pro-regenerative phenotype" by Joana Capote and colleagues, adds support to the idea that osteopontin inhibitors could be used to treat patients with Duchenne muscular dystrophy (DMD).

DMD is a progressive, and ultimately fa"



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» Summit Reports Positive Interim Data from Phase 1 Testing a New Formulation of SMT C1100 in DMD Patients Action Duchenne

» Summit Reports Positive Interim Data from Phase 1 Testing a New Formulation of SMT C1100 in DMD Patients Action Duchenne: "Today, we announced preliminary interim results from an ongoing Phase 1 trial of a new formulation of SMT C1100. As you’ll see in the release pasted below, we just announced data from the first dose in patients (with up to 3 doses planned) and from healthy volunteers. The data are encouraging and the trial is proceeding to the next dose in patients. This new formulation Phase 1 trial is a separate trial to the Phase 2 PhaseOut DMD trial that we’ve been discussing recently. Some questions that you may have:"



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New Biomarkers for Utrophin Protein Levels Likely to Aid DMD Therapy Now in Clinical Testing - Muscular Dystrophy News

New Biomarkers for Utrophin Protein Levels Likely to Aid DMD Therapy Now in Clinical Testing - Muscular Dystrophy News: "Summit Therapeutics plc announced the publication of a study into new imaging techniques that appear to reliably and reproducibly measure utrophin protein levels and muscle fiber regeneration in muscle biopsies in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) patients.

The study is important to the company because one of its lead products, now in clinical testing, is SMT C1100, a small molecule utrophin modulator that has been shown in a mice model of DMD to increase utrophin in skeletal and diaphragm muscle, leading to a significant decrease in disease pathology and an improved functional benefit. Utrophin up-regulation is considered a possible therapeutic option for all DMD patients, regardless of their underlying dystrophin mutation."



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Summit Therapeutics Supports Ninth Annual Rare Disease Day - NASDAQ.com

Summit Therapeutics Supports Ninth Annual Rare Disease Day - NASDAQ.com: "OXFORD, UK, Feb. 29, 2016 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (LSE:SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD') and Clostridium difficile infection, announces today its support for the ninth annual Rare Disease Day on 29 February 2016. The Rare Disease Day 2016 theme, Patient Voice, recognises the crucial role that patients play in voicing their needs and in initiating change that improves their lives and the lives of their families and carers.


"On this ninth annual Rare Disease Day, we at Summit take this opportunity to pay tribute to all patients and their families living with rare diseases," commented Glyn Edwards, Chief Executive Officer of Summit Therapeutics.  "The patient and parent voice is playing a vital role in the development of our utrophin modulator therapies that offer the potential to treat all boys and men living with the rare disease, DMD, and Summit has an unwavering commitment towards bringing hope for all those affected by this disease.""



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Leukaemia drug could slow progression of Duchenne muscular dystrophy

Leukaemia drug could slow progression of Duchenne muscular dystrophy: "A drug commonly used to treat leukaemia is showing potential as a treatment that could slow the progression of the muscle-wasting condition, Duchenne muscular dystrophy.

Duchenne muscular dystrophy most commonly affects boys, with around 2,400 people in the UK affected by the condition. There is currently no cure and most patients are not expected to live past the age of 30.

Researchers at the University of Sheffield investigated a drug called dasatinib, which works by blocking certain chemical signals that stimulate the growth of cancer cells. They found the same drug will also switch off similar signals in a protein implicated in Duchenne Muscular Dystrophy (DMD). This protein, called dystroglycan, has a part to play in maintaining healthy muscle tissue."



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New robotic arm could support daily activities of patients with Duchenne Muscular Dystrophy

New robotic arm could support daily activities of patients with Duchenne Muscular Dystrophy: "Researchers from the University of Twente's MIRA research centre, together with the VUmc, TU Delft and the Radboud umc, have developed the A-Gear: a robotic arm that can support the daily activities of people suffering the muscular disease Duchenne Muscular Dystrophy. They recently put the final touches to the first prototype for a discreet, body-connected support aid that can be worn under the clothing and that can support independent operation of the arm during important daily activities. "



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Summit Therapeutics gets nod for 'PhaseOut DMD' trial - Proactiveinvestors (UK)

Summit Therapeutics gets nod for 'PhaseOut DMD' trial - Proactiveinvestors (UK): "Drug developer Summit Therapeutics (NASDAQ:SMMT, LON:SUMM) has received regulatory clearance to initiate a Phase 2 proof of concept clinical trial of its SMT C1100 candidate.

The “PhaseOut DMD” trial will be on patients with Duchenne Muscular Dystrophy (DMD), a muscle wasting disorder that affects boys and young men.

It aims to provide proof of concept for SMT C1100 and utrophin modulation through measurements of muscle fat infiltration, as well as measuring utrophin protein and muscle fibre regeneration in muscle biopsies.

SMT C1100 is an orally administered, small molecule utrophin modulator that the company believes has the potential to treat all boys and young men with DMD, regardless of their underlying dystrophin gene mutation. Utrophin is functionally and structurally similar to dystrophin, a protein that is is essential for the healthy function of muscles.

The 48-week open-label trial is expected to enrol up to 40 boys ranging in age from their fifth to their tenth birthdays at sites in Europe and the US, though trials in the latter will require the US Food and Drug Administration's (FDA) approval."



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