Wednesday, 25 March 2015

» Breaking News – Translarna Update Action Duchenne

» Breaking News – Translarna Update Action Duchenne: "Contrary to announcements currently circulating, NHS England have provided no guarantees to offer a funding decision on Translarna in May.

Whilst it is true that George Freeman MP and Norman lamb MP have written to NHS England pushing for a decision in May (efforts we wholeheartedly support) Anthony Prudoe, commissioner from NHS England has just announced at the APPG inquiry report launch that there are no guarantees Translarna will be subject to a funding decision in May."

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Thursday, 19 March 2015

PPMD's 2015 Connect Conference - Parent Project Muscular Dystrophy

PPMD's 2015 Connect Conference - Parent Project Muscular Dystrophy: "Parent Project Muscular Dystrophy’s Annual Connect Conference is a unique convergence of industry partners, scientific leaders, medical providers, people living with Duchenne, and their families. This exceptional meeting has grown to be recognized worldwide as the foremost Duchenne muscular dystrophy meeting. But more than that, it is a way for families affected by Duchenne to connect with each other. To build support networks. To realize no one is on their own in the fight to end Duchenne."

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Spring Forward: Bringing the MD-CARE Act to Life - PPMD Community

Spring Forward: Bringing the MD-CARE Act to Life - PPMD Community: "Despite great odds in a political climate that was stacked against us – we achieved what was considered to have been impossible. After months of outreach to congressional champions and visits to Capitol Hill, the MD-CARE Act Amendments were passed and signed into law in the end of 2014.
Now what? Well… now the real work of implementation actually begins.
But the truth is that our community has so successfully built champions and partners across the federal agencies over the last two decades, that the implementation of the MD-CARE Amendments started long before the bill ever passed. Our federal partners ‘anticipated’ its passage and began to implement aspects of what was being called for in the new law, even before Congress was telling them they had to."

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What is Epicatechin? - PPMD Community

What is Epicatechin? - PPMD Community: "Epicatechin is a flavonoid found in dark chocolate harvested from the cacao tree. What’s that got to do with Duchenne? Epicatechin is one of dozens of flavonoids found in dark chocolate, but it is unique with respect to its effects on muscle and heart. Epicatechin has been demonstrated in animals and humans to increase the production of new mitochondria in heart and muscle (termed “mitochondrial biogenesis”) while concurrently stimulating the regeneration of muscle tissue. The progression of muscle weakness in Duchenne muscular dystrophy is associated with a loss of mitochondria, which power the skeletal muscle. Recent evidence indicates that Epicatechin is acting as a mimic of a newly discovered human hormone released by normal muscles after exercise that may be responsible for mitochondria biogenesis and stronger muscle fibers that occur normally after exercise training. Other closely related flavonoids in chocolate either have no effect or are actually inhibitory to mitochondrial biogenesis. Thus use of extracts of cacao or non-pharmaceutical grade Epicatechin may be ineffective. The current studies in muscular dystrophy are being conducted with pharmaceutical grade Epicatechin, free of any other flavonoids or contaminants. There is a trial in Europe evaluating a related compound, epigallocatechin gallate (EGCG) for its anti-oxidant effects in muscular dystrophy. ECGC has none of the mitochondrial or muscle effects that has been observed for Epicatechin, because it is the wrong shape to mimic the human hormone."

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Tuesday, 17 March 2015

Recall Notice for Deflazacort 30mg - PPMD Community

Recall Notice for Deflazacort 30mg - PPMD Community: "PPMD has been in touch with Masters Pharmaceuticals regarding a recall that was sent to many families regarding a recent shipment of Deflazacort. Here is what they told us:
The recall is specific to Deflazacort 30mg, (Marketing Authorisation Holder: Sandoz) and was recently issued by the Spanish Agency of Medicine. The batches that are affected and subject to the recall notification are J021 (expiry 30/06/2017) and J028 (expiry 30/09/2017). Masters is currently trying to establish the reason for the recall but have no further information at this time, but they are working with the Products manufacturer. They are contacting ~230 customers who purchased the affected batches however, we believe that most will only have batch J028 remaining."

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Thursday, 5 March 2015

Important New Duchenne Steroid Protocol: Download the "PJ Nicholoff Steroid Protocol" - PPMD Community

Important New Duchenne Steroid Protocol: Download the "PJ Nicholoff Steroid Protocol" - PPMD Community: "Phillip James “PJ” Nicholoff was a vibrant, 31-year-old man living with Duchenne muscular dystrophy. He had an encyclopedic love of all college and professional sports; he and his family traveled all over the country to catch games. He had a degree in web design and worked his entire adult life. He enjoyed swimming, dating, and driving his own car – much like any other 31-year-old young man.
PJ had been treated with daily corticosteroids since the age of 6. He endured several pathologic fractures, likely a result of his steroid treatment and his non-ambulatory status. In November 2013, on his last trip to Florida, he fractured his humerus and hip, and was transported by plane to a hospital closer to his northern home. PJ had orthopedic surgery to manage both of these fractures. After these repairs, he developed respiratory distress, shock, and died. Review of the medical record suggested that he had not received consistent corticosteroid replacement during his hospitalizatio"

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Parent Project Muscular Dystrophy & Foundation to Eradicate Duchenne Award $1 Million Grant to ReveraGen BioPharma - PPMD Community

Parent Project Muscular Dystrophy & Foundation to Eradicate Duchenne Award $1 Million Grant to ReveraGen BioPharma - PPMD Community: "Parent Project Muscular Dystrophy (PPMD) is excited to join forces with Foundation to Eradicate Duchenne (FED) to award ReveraGen BioPharma (ReveraGen) a $1 million grant to help fund the chronic toxicology program for VBP15, a combined action drug that addresses multiple levels of Duchenne."

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Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (And Were Not Afraid to Ask) - PPMD Community

Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (And Were Not Afraid to Ask) - PPMD Community: "This webinar will review the basic biology of dystrophin (the gene, structure-function of the protein, its role in the dystrophin-glycoprotein complex, characteristics of different forms of dystrophin (both naturally occurring and drug-induced truncated forms), discuss how dystrophin is measured in both preclinical studies and clinical trials, and discuss the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. We will also discuss what is required in order to have dystrophin considered as a surrogate."

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