Expanding the Duchenne Therapeutic Opportunities by Targeting Myostatin - PPMD Community

Expanding the Duchenne Therapeutic Opportunities by Targeting Myostatin - PPMD Community: "We all understand that Duchenne muscular dystrophy is characterized by muscle wasting and associated loss of function and that there are considerable efforts underway to develop drugs and biologics (cell and gene therapy) to address the primary problem in Duchenne—the absence of dystrophin. Restoring dystrophin or replacing dystrophin with replacement protein are considered foundational therapies.
 
That said, the Duchenne community is well aware of the need for combination therapies. Combinations would include compounds that target what is referred to as the ‘downstream pathology’ or the changes that occur because dystrophin is absent. This includes anti-inflammatories, anti-fibrotics, factors that control muscle regeneration and fiber size, compounds that improve circulation to muscle, and compounds that improve mitochondrial function (mitochondria are considered the powerhouses of cells). We are all hopeful that by combining several of these targeted therapies, we could end Duchenne, stop progression for every individual. This is the dream of precision or personalized medicine. The right drug, at the right time, in the right dose for the right person. It requires planning and it will require combinations."



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Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner

Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner: "Statins are widely known for their use in improving cardiovascular health through lowering blood cholesterol levels. What prompted you to study their use in Duchenne muscular dystrophy (DMD)?"



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» Translarna Update: Action Duchenne submit FOI Requests to HM Treasury and the Department of Health Action Duchenne

» Translarna Update: Action Duchenne submit FOI Requests to HM Treasury and the Department of Health Action Duchenne: "Translarna Update: Action Duchenne submit FOI Requests to HM Treasury and the Department of Health
Posted on: July 20th, 2015 | 1 comment

Action Duchenne have submitted Freedom of Information (FOI) requests to both Her Majesty’s Treasury and the Department of Health as part of the ongoing campaign for access to Translarna."



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Study finds that muscle fibrosis inhibition causes increases in utrophin levels and revertant myofibers in Duchenne Muscular Dystrophy

Study finds that muscle fibrosis inhibition causes increases in utrophin levels and revertant myofibers in Duchenne Muscular Dystrophy: "In a recent study published in Oncotarget, researchers found an inverse correlation between the level of muscle fibrosis, utrophin and the number of revertant myofibers in Duchenne Muscular Dystrophy (DMD).

Results from this study reveal common links between the fibrotic and utrophin-synthesis pathways and offer new insights into the regulation of utrophin synthesis in Duchenne Muscular Dystrophy."



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» PTC Therapeutics fundraising endeavours for Action Duchenne Action Duchenne

» PTC Therapeutics fundraising endeavours for Action Duchenne Action Duchenne: "PTC Therapeutics are sponsoring the LEJOG 2015 Challenge, which takes place between 26th July and 4 August.

Action Duchenne has 21 riders taking part in LEJOG 2015, including three relay teams. If you would like to support the challenge in fundraising or get involved in a similar event please contact Paul Bown via 020 8556 9955 or paulbown@actionduchenne.org

Lands End to John O’Groats is one of the most iconic cycle tours around , taking in some of the most spectacular scenery in the UK. It’s a challenging event that will see participants pedal the length of the country over 10 days, averaging almost 100 miles a day.



 

Secondly twelve members of staff from PTC Therapeutics are running the New York Marathon on 1 November 2015 for Action Duchenne. They work for the company across the world in the following countries – Brazil, USA, France, Germany, Switzerland and the UK.

The TCS New York City Marathon is the world’s largest marathon, with more than 50,000 finishers in 2013. It began in 1970 with a small group of runners in Central Park and has been held every year since, with the exception of 2012, when the race was cancelled due to Hurricane Sandy. The course starts in Staten Island and passes through all five boroughs of the city."



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» German Federal Joint Committee Issues Positive Medical Benefit Rating for Translarna™ in Duchenne Action Duchenne

» German Federal Joint Committee Issues Positive Medical Benefit Rating for Translarna™ in Duchenne Action Duchenne: "PTC Therapeutics, Inc today announced that the Benefit Assessment by Germany’s Federal Joint Committee (G-BA) indicated that Translarna™ (ataluren) provided a benefit for ambulatory patients aged five years and older with nonsense mutation Duchenne Muscular Dystrophy (nmDMD). The G-BA came to the decision that the existing clinical data package presented by PTC provided convincing evidence for the demonstration of a clinically meaningful added benefit. PTC received a 3 in the rating system established under the German pharmaceutical law.

“We are pleased by the positive assessment we received from the G-BA for Translarna. It reflects the medical benefit provided for nonsense mutation DMD patients where there are no other approved therapies,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “We appreciate the G-BA’s recognition of Translarna’s benefit for these patients with such high unmet medical need.”"



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» Sarepta Therapeutics announce plans to submit rolling NDA for eteplirsen Action Duchenne

» Sarepta Therapeutics announce plans to submit rolling NDA for eteplirsen Action Duchenne: "Sarepta Therapeutics, Inc. announced that the Company held a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) regarding eteplirsen. Sarepta has agreed with the Agency to initiate a rolling NDA submission and will submit the non-clinical: chemistry, manufacturing and controls components of the NDA by the end of this week. As previously announced, Sarepta plans to submit the final component of the NDA by mid-year 2015.
“We will initiate a rolling NDA submission to facilitate the regulatory review of the NDA,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “The initiation of our NDA submission for eteplirsen marks a significant milestone for the Duchenne community and we look forward to completing the submission by the middle of the year and to continuing to work with the Agency towards the goal of providing treatments to patients as quickly as possible.”
You can read the full press release from Sarepta by opening the document below:"



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