SLU scientists provide promising approach in designing new drugs for DMD

SLU scientists provide promising approach in designing new drugs for DMD: "In a paper published in the Nature journal Scientific Reports, Saint Louis University (SLU) researchers report that a new drug reduces fibrosis (scarring) and prevents loss of muscle function in an animal model of Duchenne muscular dystrophy (DMD), providing a promising approach in designing new medications for those suffering from DMD."



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New initiative aims to improve chances of medicines receiving positive HTA decisions

New initiative aims to improve chances of medicines receiving positive HTA decisions: "A unique initiative launched today by muscular dystrophy charity Duchenne UK is set to radically simplify the way necessary evidence is generated for submissions made to health technology appraisals (HTA) bodies such as the National Institute for Health and Care Excellence (NICE) and the Haute Autorité de Santé (HAS).

Launched today at the 8th World Orphan Drug Congress in Barcelona, this innovative collaboration – entitled HERCULES –  will focus on medicines used to treat Duchenne muscular dystrophy (DMD).  It will be the first time a group of pharmaceutical industry partners have ever collaborated to generate, share and align disease level data across an entire condition.

Although this new initiative focuses on DMD, it paves the way for similar approaches in other rare diseases, and has the potential to improve the chances of medicines in rare and orphan diseases receiving positive HTA decisions.  This unique collaborative approach has been welcomed by a range of industry health economists, as well as former NICE personnel."



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Summit completes initial dosing trials on Duchenne muscular dystrophy drug - Pharmaceutical Business Review

Summit completes initial dosing trials on Duchenne muscular dystrophy drug - Pharmaceutical Business Review: "Summit Therapeutics has completed the half-way stage of dosing of its Duchenne Muscular Dystrophy (DMD) candidate ezutromid in a 48-week phase 2 trial, dubbed PhaseOut DMD.

The UK-based Summit is likely to report results from the initial 24-week dosing period in the first quarter of 2018 which are anticipated to contain data from muscle biopsies, MRI and functional tests, and also safety data.

Summit Therapeutics chief operating officer and medical officer David Roblin said: "In the interim data readout, we aim to show a positive change following ezutromid treatment in muscle structure and health through the evaluation of muscle biopsies.

“This could potentially provide the first evidence of proof of mechanism for utrophin modulators in patients and give hope to those living with DMD.”"



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Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients: "After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients' hearts appeared improved, results from a new study show. Patients in the study also scored higher on arm strength tests after receiving the cell infusions.

Results from the HOPE Duchenne randomized clinical trial of 25 patients were presented today at the American Heart Association Scientific Sessions in Anaheim. The cardiac progenitor cells administered to the patients were contained in CAP-1002, the lead investigational therapy under development at Capricor Therapeutics, Inc. (NASDAQ: CAPR)."



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DMD Study Shows Protein Utrophin Reduces Mitochondria Damage

DMD Study Shows Protein Utrophin Reduces Mitochondria Damage: "In recent years scientists have learned that the protein utrophin can substitute for the dystrophin protein that is missing in muscular dystrophy.

Researchers have now discovered that utrophin can reduce damage to energy-producing cell components known as mitochondria, whose deterioration is linked to MD. The reduction in damage improved the muscle health of mice with Duchenne muscular dystrophy.

Summit Therapeutics and collaborators from the University of Oxford presented the findings at the 15th Action Duchenne International Conference 2017 in Birmingham, England, Nov. 10-12."



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Duchenne MD Exon-skipping Therapy Is Now in Phase 1 Trial

Duchenne MD Exon-skipping Therapy Is Now in Phase 1 Trial: "Wave Life Sciences has started a Phase 1 clinical trial of a treatment for a gene malfunction linked to the most common form of Duchenne muscular dystrophy."



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Summit Highlights Utrophin Modulation as a Potential Universal Treatment Option in DMD at Action Duchenne Nasdaq:SMMT

Summit Highlights Utrophin Modulation as a Potential Universal Treatment Option in DMD at Action Duchenne Nasdaq:SMMT: "Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM) the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, announces that the Company and collaborators from the University of Oxford will highlight the potential of utrophin modulation as a universal treatment for DMD in several presentations at the 15th Action Duchenne International Conference 2017, taking place November 10-12, 2017, in Birmingham, UK. The conference brings together families living with Duchenne and Becker muscular dystrophy, along with experts and industry members aiming to treat these diseases.

The Company's scientific adviser and co-founder, Professor Kay Davies from the University of Oxford, will present recently published preclinical data highlighting the benefits of utrophin modulation on muscle health in animal models of DMD. The data show that continuously expressing utrophin in a dystrophin-deficient background can reduce mitochondrial aberration and oxidative stress. Aberrant mitochondria drive, in part, oxidative stress, which is a contributing factor to muscle damage in DMD. In addition, Professor Davies is also expected to discuss previously published preclinical data of utrophin modulators, including ezutromid, that have shown these compounds' potential to prevent molecular disease, leading to functional improvements in mdx mice."



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DMD Therapy Exondys 51 Gets a Hand from Immune Cells, Study Reports

DMD Therapy Exondys 51 Gets a Hand from Immune Cells, Study Reports: "The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice."



The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice.

CRISPR-Gold corrects gene mutations in mice with Duchenne muscular dystrophy

CRISPR-Gold corrects gene mutations in mice with Duchenne muscular dystrophy: "Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne muscular dystrophy, a severe muscle-wasting disease. A new study shows that a single injection of CRISPR-Gold, as the new delivery system is called, into mice with Duchenne muscular dystrophy led to an 18-times-higher correction rate and a two-fold increase in a strength and agility test compared to control groups."



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Summit Therapeutics PLC teams up with cTAP as it looks to accelerate development of its DMD drug

Summit Therapeutics PLC teams up with cTAP as it looks to accelerate development of its DMD drug: "Drug discoverer Summit Therapeutics PLC (LON:SUMM; NASDAQ:SMMT) has teamed up with the Collaborative Trajectory Analysis Project to try and accelerate the development of its lead Duchenne muscular dystrophy candidate.

Summit’s utrophin modulator for the treatment of DMD – ezutromid – is currently in a phase II clinical trial, with data from the 48-week study expected in the third quarter of 2018.

But the dual-listed firm hopes that by partnering with cTAP, it can potentially adapt and improve the overall clinical process."



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