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Action Duchenne launches new Research Strategy for Duchenne muscular dystrophy

Posted on: November 17th, 2017 | 0 comments

  Action Duchenne, a leading UK-wide patient and parent-led organisation for Duchenne Muscular Dystrophy, is delighted to announce the publication of a new 3 year research strategy. Duchenne muscular dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births (around 2500 people have DMD

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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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Astellas acquires Mitobridge under existing collaboration

Posted on: December 11th, 2017 | 0 comments

Astellas has exercised its exclusive option right to acquire Mitobridge under the amended terms of the October 2013 partnership agreement governing their an R&D collaboration focused on discovering and developing novel drugs that target mitochondrial function. Mitobridge will become a wholly-owned subsidiary of Astellas following the close of the acquisition. Under the 2013 partnership agreements,

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FDA new approach would reduce number of patients treated with placebo

Posted on: December 11th, 2017 | 0 comments

The U.S. Food and Drug Administration (FDA) has published its recommendation for a new approach to drug development for rare pediatric diseases. The new approach supports the FDA’s commitment to increase the efficiency and speed of developing drugs, as well as supporting the sponsors of the research. The draft guidance, ‘Pediatric Rare Diseases: Collaborative Approach

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Results announced from survey ‘Juggling care and daily life’

Posted on: December 11th, 2017 | 0 comments

The 17th October 2017 was an important date for the rare disease community.  The highlights of the results of the first Eurordis (Rare Diseases Europe) rare disease survey, were presented at an event held at the European Parliament in Brussels. Over 3,000 rare disease voices across Europe responded to the survey ‘Juggling care and daily life: The balancing

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Government and Life Sciences sector agree sector deal

Posted on: December 6th, 2017 | 0 comments

Business Secretary Greg Clark and Health Secretary Jeremy Hunt have announced that the Government and Life Sciences sector have agreed a “transformational sector deal”. Key themes of the deal: The deal sets out a plan for key priorities for the sector going forward, with a vision and strategy that are aligned to the pillars of

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Hanging on every word – reflections from the 2017 Action Duchenne Conference

Posted on: December 6th, 2017 | 0 comments

One of our wonderful Parents, Lizzie Deeble has published a guest post on ‘Conference Inference’. Lizzie Deeble writes about her first experience at a conference, where she found she was attending as a parent, a carer, and an individual in her own right. “During the weekend of the 10th-12th of November 2017, exactly 13 months

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Research Updates

Posted on: December 4th, 2017 | 0 comments

Duchenne muscular dystrophy research continues at pace:   —Researchers devise improved gene-editing process for Duchenne muscular dystrophy— The MedicalXpress website reports that Dr. Eric Olson, Director of the Hamon Center for Regenerative Science and Medicine and Co-Director of the Wellstone Muscular Dystrophy Cooperative Research Center at UT Southwestern have developed an imporved gene-editing process for Duchenne.

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Solid initiate new clinical trial for 4-17 year olds living with Duchenne

Posted on: December 1st, 2017 | 0 comments

Solid Biosciences has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne muscular dystrophy. The Phase I/II adaptive study, called IGNITE DMD, will evaluate the safety and efficacy of a single intravenous (IV) dose of SGT-001 in ambulatory and non-ambulatory adolescents and children with Duchenne. Enrolment

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Capricor to launch HOPE-2 study of Cap-1002 for young people with advanced Duchenne

Posted on: December 1st, 2017 | 0 comments

Capricor has received a green light from the U.S. Food and Drug Administration to launch a Phase 2 study of CAP-1002 cell therapy in children and adults with advanced Duchenne muscular dystrophy. Capricor expects to open the trial to patient enrolment in early 2018 and, if successful, to begin steps needed to receive FDA approval of CAP-1002 as

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Action Duchenne are Recruiting

Posted on: December 1st, 2017 | 0 comments

We are looking for a Challenge Events Fundraising Officer to join our team in London. Candidates should be enthusiastic, energetic and able to manage a varied workload. A knowledge of, and passion for, helping people with Duchenne are desirable qualities so we are very keen to hear from those in the Duchenne community. You can find the job description

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Capricor Therapeutics receive green-light from FDA to conduct Duchenne trial

Posted on: November 30th, 2017 | 0 comments

  —Capricor Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for CAP-1002— —Potential Registration Trial in Duchenne Muscular Dystrophy on Track to Initiate in First Quarter of 2018—   Capricor Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to conduct a new

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Action Duchenne launches new Research Strategy for Duchenne muscular dystrophy

November 17th, 2017

  Action Duchenne, a leading UK-wide patient and parent-led organisation for Duchenne Muscular Dystrophy, is

Tell me more

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

FDA new approach would reduce number of patients treated with placebo

December 11th, 2017

The U.S. Food and Drug Administration (FDA) has published its recommendation for a new approach

Tell me more

Results announced from survey ‘Juggling care and daily life’

December 11th, 2017

The 17th October 2017 was an important date for the rare disease community.  The highlights of the

Tell me more

Duchenne – How do you measure a life?

Living with Duchenne – an animation

Taking charge of Duchenne

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