» Published brief of changes to Disabled Facilities Grant Funding. Action Duchenne

» Published brief of changes to Disabled Facilities Grant Funding. Action Duchenne: "This month heralds a significant change in the way that national funding for disabled facilities grants is paid, and Care and Repair England have published their guidelines. This is aimed for those who, plan, commission and provide health, social care and housing related provision.

In short, instead of national government making direct payment to each local housing authority to help them to meet the cost of providing Disabled Facilities Grants, this allocation will, for the first time, be paid through Better Care Funds. These bodies will then pass on the allocation to the relevant Housing Authority.

To read these guidelines outlining the change in procedure, click the following link:

Disabled Facilities Grant Funding Changes"



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» Santhera’s Positive Phase III Trial in Patients with DMD Published in The Lancet Action Duchenne

» Santhera’s Positive Phase III Trial in Patients with DMD Published in The Lancet Action Duchenne: "The results of the DELOS trial demonstrated that Raxone/Catena significantly reduced the annual decline in Peak Expiratory Flow (PEF as percent predicted, PEF%p) by 66% compared to patients taking placebo. Other respiratory function endpoints such as Forced Vital Capacity (FVC) and Forced Expiratory Volume (FEV1) corroborated these results and showed a consistent pattern with treatment differences supporting efficacy of Raxone/Catena over placebo in the preservation of respiratory function. Researchers concluded that Raxone/Catena represents a new treatment option for DMD patients."



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» Santhera Pharmaceuticals receives FDA fast track designation for Raxone/Catena Action Duchenne

» Santhera Pharmaceuticals receives FDA fast track designation for Raxone/Catena Action Duchenne: "Santhera Pharmaceuticals receives FDA fast track designation for Raxone/Catena (idebenone) for the treatment of Duchenne Muscular Dystrophy.

“We are very pleased that the FDA has granted Fast Track designation for Raxone/Catena which further underscores the unmet medical need for effective treatments for patients with DMD” commented Thomas Meier PhD CEO of Santhera. “On the basis of the positive data from our Phase III trial with Raxone/Catena in DMD we have started to prepare a New Drug Application and plan to meet with the FDA in the coming weeks to discuss our NDA dossier in a pre-NDA meeting.”More: http://www.pharmiweb.com/pressreleases/pressrel.asp?ROW_ID=112438#.VSaOvSLF8bU#ixzz3Wp8uen6L"



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» Researchers Publish Valuable Data on Duchenne Muscular Dystrophy Progression Using the 6-Minute Walk Test Action Duchenne

» Researchers Publish Valuable Data on Duchenne Muscular Dystrophy Progression Using the 6-Minute Walk Test Action Duchenne: "In a recent study titled “Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes,” a research team from the Department of Pediatric Neurology at the Catholic University in Rome, Italy examined the changes at 3 years follow-up in a 6-minute walk test in patients Duchenne muscular dystrophy, the most used primary outcome measure in international multi center clinical trials for patients with the disease."



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» New exon-skipping strategy rescues dystrophin Action Duchenne

» New exon-skipping strategy rescues dystrophin Action Duchenne: "Antisense oligonucleotides (AONs) hold promise for therapeutic correction of many genetic diseases via exon skipping, and the first AON-based drugs have entered clinical trials for neuromuscular disorders. However, despite advances in AON chemistry and design, systemic use of AONs is limited because of poor tissue uptake, and recent clinical reports confirm that sufficient therapeutic efficacy has not yet been achieved. Here they present a new class of AONs made of tricyclo-DNA (tcDNA), which displays unique pharmacological properties and unprecedented uptake by many tissues after systemic administration."



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