PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC begins rolling NDA submission for Translarna to treat nmDMD: "PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has commenced a rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). A rolling submission allows completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis. PTC expects to finalize the application in the fourth quarter of 2015 following the completion of the ACT DMD confirmatory Phase 3 clinical trial."



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» Sarepta Thereaputics release information on trial for SRP-4053 for skipping exon 53 Action Duchenne

» Sarepta Thereaputics release information on trial for SRP-4053 for skipping exon 53 Action Duchenne: "Sarepta Therapeutics have released information regarding the clinical trial for their drug candidate SRP-4053 for skipping exon 53, which is taking place in Europe.

Enrolment for the London centre at Great Ormond Street Hospital is currently taking place, with the Newcastle Centre planned for the future.
 
Eligibility criteria and other information is included in the posting on the clinical trials website.

 

Inclusion criteria includes:

 

being amenable to exon 53 skipping,
stable pulmonary and cardiac function,
minimum performance on 6 minute walk test, North Star Ambulatory Assessment, and rise (Gowers) test as specified in the study protocol,
On a stable dose of corticosteroids for at least 6 months.
Aged between 6-15 years old
Intact right and left biceps muscles or an alternative upper arm muscle group."



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» European early access programme for Carmeseal-MD(TM) Action Duchenne

» European early access programme for Carmeseal-MD(TM) Action Duchenne: "Phrixus Pharmaceuticals aims to demonstrate that Carmeseal-MD has beneficial effects in patients with Duchenne and Becker muscular dystrophy by delivering an improvement in cardiac and respiratory function via protection of heart muscle and diaphragm.   The drug acts as a molecular band-aid by binding to, and then sealing, microscopic tears in muscle cells caused by the lack of functional dystrophin.  This prevents the uncontrolled leakage of calcium which in turn increases the performance of heart muscle and diaphragm and prevents their degeneration."



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» NHS Delays Decision on Translarna Action Duchenne

» NHS Delays Decision on Translarna Action Duchenne: "Action Duchenne has been informed that NHS England has delayed the CPAG meeting that was due to take place next week on the 9th of December. The meeting has been cancelled as NHS England are re-considering how it assesses new treatments.

We are extremely concerned and disappointed about this decision. You will recall that from our conference that the complex and overly long commissioning process for new treatments was called into question by our CEO. This delay of the CPAG is symptomatic of the fragile commissioning process."



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» Please sign petition for full funding Translarna Action Duchenne

» Please sign petition for full funding Translarna Action Duchenne: "We would like to ask that our members support & sign the petition below for each of the UK NHS authorities to fully fund the costs for Translarna, which would be the first treatment for Duchenne to become available to patients in the UK.

Action Duchenne’s staff have been working for the previous 3 months with NHS England, PTC Therapeutics and other UK Duchenne charities to ensure that those who are in the decision making process have been made fully aware of the importance of funding of this first treatment for Duchenne."



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