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Long-term microdystrophin pre-clinical work, moving towards a potential therapy restores muscle function in canines

Posted on: July 26th, 2017 | 3 comments

Promising microdystrophin pre-clinical work, moving towards a potential therapy has shown to restore muscle function in the canine model (dogs). Golden Labradors are naturally affected by Duchenne muscular dystrophy so researchers chose them for mammalian trials, ahead of tests in humans. The new pre-clinical research carried out by researchers at the Royal Holloway in London and French

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First draft of agenda announced – Action Duchenne International Conference

Posted on: June 28th, 2017 | 0 comments

We are extremely excited to announce the first draft of the agenda for the Action Duchenne International Conference 2017. The agenda is liable to change but gives a good picture of the breadth and depth of content at the event. The conference will take place Friday 10 to Sunday 12 November 2017 at the Hilton

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GTx Update on Preclinical Studies

Posted on: August 15th, 2017 | 0 comments

GTx have reported financial results for the second quarter of 2017 and highlighted recent accomplishments and upcoming milestones. Enobosarm in Duchenne muscular dystrophy (DMD) The Company has conducted preclinical studies evaluating selective androgen receptor modulator (SARMs) in Duchenne. In preclinical models of Duchenne, GTx SARMs have increased body weight, lean mass and physical function. Click here for

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NICE Consultation on Guidelines for Suspected Neurological Conditions

Posted on: August 14th, 2017 | 0 comments

NICE (the National Institute for Health and Care Excellence) have released for consultation, a new set of draft guidelines for healthcare professionals to use in recognising and referring suspected cases of neurological conditions (including Duchenne and Becker muscular dystrophy). Duchenne muscular dystrophy is included in the draft guidelines (pages 131-136) under the ‘Motor development delay

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Test with the Potential to More Reliably Screen for Duchenne in Newborns is Developed

Posted on: August 11th, 2017 | 0 comments

Researchers at Cardiff University, in collaboration with biotechnology company PerkinElmer, have created a more reliable test to screen newborn babies for Duchenne muscular dystrophy. Together they have developed a diagnostic kit that can analyse neonatal dried blood spots for the presence of Duchenne.   Professor Ian Weeks, Dean of Clinical Innovation and Head of Cardiff University’s

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Pharmaceutical Companies Working in Duchenne Report Q2 Financial Results and Provide Business Updates

Posted on: August 11th, 2017 | 0 comments

—A number of pharmaceutical and biotech companies working in the field of Duchenne muscular dystrophy, have recently reported their 2nd quarter financial results and provided updates on their business activities and programmes.— First to update the community was PTC Therapeutics on 8 August. “Since our founding nearly 20 years ago, it has been our mission

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Worldwide licence for new drug SOMO266 as SOM Biotech expands to the United States

Posted on: August 9th, 2017 | 0 comments

The med-tech biopharmaceutical company, SOM Biotech, has granted a licence agreement with Corino Therapeutics Inc. for its first product SOMO266 for the treatment of TTR Amyloidosis following encouraging Phase 2 study results. SOM Biotech is a med-tech biopharmaceutical company which specialises in repurposing drugs (using drugs which already exist on the market) for the treatment

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Mitobridge’s Potential Treatment for Duchenne Advances into Clinical Development

Posted on: August 8th, 2017 | 0 comments

Mitobridge, Inc., a pioneer in the discovery and development of products that improve mitochondrial function, today announces a key milestone with the initiation of the first-in-human Phase I trial of its PPAR-delta (PPARd) modulator, MA-0211 (also known as MTB-1). The study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of MA-0211 in healthy volunteers, which

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Pharrell Williams scam

Posted on: July 31st, 2017 | 0 comments

We wanted to alert the wider community that the condition Duchenne muscular dystrophy has been used in a scam, but someone posing as Pharrell Williams. If you are contacted by a profile on social media posing as Pharrell Williams, please be aware that they are using Duchenne to solicit funds. We advise contacting your local police non-emergency

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Sarepta announces its partner, Genethon, published new micro-dystrophin gene therapy data

Posted on: July 28th, 2017 | 0 comments

Data show for the first time a systemic therapeutic effect in DMD dogs using a rAAV2/8 micro-dystrophin gene therapy approach without immunosuppressive treatment Sarepta has announced the publication of data validating Genethon’s micro-dystrophin gene therapy approach in an animal model for Duchenne muscular dystrophy. Click here for our article. The results were featured in the July

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Highlights from muscle-bone meeting co-funded by Action Duchenne

Posted on: July 26th, 2017 | 0 comments

Muscle-Bone Deficits In Chronic Neuromuscular Conditions Symposium, 15th June 2017 This successful event was held on Thursday 15th June 2017, in the Lecture Theatre at the QEUH Teaching and Learning Centre, organised by the Developmental Endocrinology Research Group and supported by Action Duchenne, the Office for Rare Conditions, and Muscular Dystrophy UK.  Over 90 registered participants were in attendance throughout the day which

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Long-term microdystrophin pre-clinical work, moving towards a potential therapy restores muscle function in canines

July 26th, 2017

Promising microdystrophin pre-clinical work, moving towards a potential therapy has shown to restore muscle function

Tell me more

First draft of agenda announced – Action Duchenne International Conference

June 28th, 2017

We are extremely excited to announce the first draft of the agenda for the Action

Tell me more

Sarepta announces its partner, Genethon, published new micro-dystrophin gene therapy data

July 28th, 2017

Data show for the first time a systemic therapeutic effect in DMD dogs using a

Tell me more

Duchenne – How do you measure a life?

Living with Duchenne – an animation

Taking charge of Duchenne

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