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Summit presents data from Phase 1 clinical programme of lead utrophin modulator, ezutromid

Posted on: June 22nd, 2017 | 0 comments

Summit Therapeutics has today announced the presentation of clinical data from two Phase 1 clinical trials of ezutromid, Summit’s lead utrophin modulator, at the European Paediatric Neurology Society Congress in Lyon, France. The oral presentation focussed on the Company’s positive efforts to improve absorption and increase exposure of ezutromid in patients with Duchenne muscular dystrophy, as

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Our guide to the Early Access to Medicines Scheme

Posted on: June 22nd, 2017 | 0 comments

  Yesterday the Medicines and Healthcare products Regulatory Agency (MHRA) through the Early Access to Medicines Scheme (EAMS) made a decision that due to data provided by Santhera Pharmaceuticals showing Raxone could slow down the decline in breathing difficulties for those living with Duchenne, it would approve use of Raxone through the EAMS Programme. Due

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What Raxone being given approval through the EAMS scheme means for the Duchenne community

Posted on: June 22nd, 2017 | 0 comments

  Over a number of years we have worked closely with Santhera Pharmaceuticals and other Duchenne charities to encourage them to take part in Santhera’s Phase III Clinical Trial study. We did this as we believed it was important to investigate whether Raxone (idebenone) could help meet the desires of those living with Duchenne to

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Raxone is the first Duchenne drug approved through the Early Access to Medicines Scheme

Posted on: June 22nd, 2017 | 0 comments

—We are excited to share with the Duchenne community, that Santhera Pharmaceutical’s drug Raxone, is the first Duchenne drug approved via the Early Access to Medicines Scheme (EAMS)— —The drug’s purpose is to stop respiratory decline, in people living with Duchenne who are not on steroids— Santhera has announced that the UK’s Medicines and Healthcare products

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Aquatic therapy for children with Duchenne muscular dystrophy

Posted on: June 21st, 2017 | 0 comments

Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation This feasibility study showed that, although aquatic therapy for Duchenne muscular dystrophy is valued, its delivery in tertiary centres poses problems and further intervention development is needed before a full randomised controlled trial. Background Duchenne muscular dystrophy

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Sarepta and Genethon form partnership

Posted on: June 21st, 2017 | 0 comments

Sarepta Therapeutics announced today that it is partnering with Genethon, a non-profit organisation that develops gene therapies for rare diseases. Genethon is currently developing a micro-dystrophin gene therapy, which could be a potential treatment for Duchenne muscular dystrophy. This partnership will help to advance this research by bringing together the expertise of Sarepta and Genethon.

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Action Duchenne funds pivotal work in validating a promising outcome measure

Posted on: June 21st, 2017 | 0 comments

A novel device called ActiMyo® has been proposed for its use as an outcome measure in ambulant and non-ambulant Duchenne trials. ActiMyo offers the opportunity to conceptualise shorter studies with much less patients compared with the currently available outcome measures. It is a portable device capable of providing information registered through an accelerometer, a magnetometer

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Our Duchenne Champions

Posted on: June 20th, 2017 | 0 comments

  After the election results of 8 June 2017 we now have many more #DuchenneChampions both inside and outside of Parliament. Through signing up to become #DuchenneChampions they are now committed to working with the Duchenne community across the UK to call on policies to be implemented for faster access to medicines for those living with

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Skydiving Superstars!

Posted on: June 19th, 2017 | 0 comments

8 months ago we set ourselves a challenge of trying to get as many people as we could doing a tandem parachute jump on the same day, raising lots of money to help in finding a cure for Duchenne at the same time.  We were bowled over by the response and had a brilliant 16

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Sarepta recruits for Phase 3 ESSENCE trial to test two potential therapies

Posted on: June 19th, 2017 | 0 comments

Sarepta Therapeutics is recruiting up to 99 boys with Duchenne muscular dystrophy to test two new compounds, SRP-4045 and SRP-4053, in a Phase 3 clinical trial. The ESSENCE study is a double-blind, placebo-controlled, multi-center Phase 3 clinical trial (NCT02500381) with an open-label extension period, evaluating the two drugs’ safety and efficacy in boys between 7 to 13

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Summit presents data from Phase 1 clinical programme of lead utrophin modulator, ezutromid

June 22nd, 2017

Summit Therapeutics has today announced the presentation of clinical data from two Phase 1 clinical trials

Tell me more

What Raxone being given approval through the EAMS scheme means for the Duchenne community

June 22nd, 2017

  Over a number of years we have worked closely with Santhera Pharmaceuticals and other

Tell me more

Duchenne – How do you measure a life?

Living with Duchenne – an animation

Taking charge of Duchenne

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