Friday 22 June 2012

Phrixus Pharmaceuticals, Inc. Announces $623,000 in NIH Funding for Its Program in Duchenne Muscular Dystrophy

Phrixus Pharmaceuticals, Inc. Announces $623,000 in NIH Funding for Its Program in Duchenne Muscular Dystrophy: Phrixus Pharmaceuticals, Inc., a clinical-stage, specialty pharmaceutical company focused on innovative therapies for Duchenne muscular dystrophy (DMD) and heart failure, today announced that it has received an SBIR Phase 1 award for $623,000 from the National Institutes of Health (NIH) for its grant application titled "Treatment of Muscular Dystrophy-Associated Dilated Cardiomyopathy with P-188."
"This funding constitutes validation for the potential utility of Carmeseal in DMD. We expect that it will allow us to extend administration of Carmeseal to a new, clinically relevant route of administration, subcutaneous delivery," said Thomas A. Collet, president and CEO.

Arbroath Brother has lost both his soul mates - Local Headlines - Arbroath Herald

Brother has lost both his soul mates - Local Headlines - Arbroath Herald: AN ARBROATH couple have suffered the agony of losing a second son in as many years to a debilitating disease.

John Mathieson, who was just 21 years old, died on Tuesday afternoon.

Norman and Yvonne Mathieson, McGregor’s Walk, have worked tirelessly over the years to provide a better quality of life for their three sons, all of whom suffered from Duchenne muscular dystrophy.

Carl ‘Action’ Tilson to carry Olympic Flame on to stage at Olympic Torch Party, Albert Square Manchester - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Carl ‘Action’ Tilson to carry Olympic Flame on to stage at Olympic Torch Party, Albert Square Manchester - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: CARL ‘ACTION’ TILSON TO CARRY OLYMPIC FLAME ON TO STAGE AT OLYMPIC TORCH PARTY, ALBERT SQUARE MANCHESTER
Olympic torch bearer happy ending for Carl, thanks to help from volunteers at the torch bearer hotline

AVI BioPharma and PPMD team up to run the Rock 'n' Roll Seattle Marathon - PPMD Community

AVI BioPharma and PPMD team up to run the Rock 'n' Roll Seattle Marathon - PPMD Community: Run For Our Sons�(PPMD's endurance program) is teaming up with AVI BioPharma�in Saturday's Rock 'n' Roll Seattle Marathon�to raise money and awareness to help end Duchenne!

AVI and PPMD have a long history of working together to develop treatments that may help to prolong the lives of those living with Duchenne. We have always been grateful to the dedication AVI has shown to the Duchenne community as they continue to work on treatments that will help our sons live longer, stronger lives. Now they are taking that dedication a step further by lacing up their running shoes and pounding the streets of Seattle to help raise awareness and end Duchenne.

Monday 18 June 2012

PPMD Leads Critical Letter for Rare Disease Community - PPMD Community

PPMD Leads Critical Letter for Rare Disease Community - PPMD Community: Parent Project Muscular Dystrophy (PPMD) has teamed up with�EveryLife Foundation for Rare Diseases�and�Genetic Alliance�to galvanize the rare disease community and send a strong message to Congress to ensure the most important provisions for the rare disease community were included in the final�Prescription Drug User Fee Act (PDUFA) legislation. The provisions are aimed at accelerating Food and Drug Administration (FDA) review of therapies to treat rare diseases and to enable FDA to more aggressively engage external experts in reviewing candidate therapies.

Tuesday 12 June 2012

New Cardiomyopathy Study for DMD Recruiting in Ohio

New Cardiomyopathy Study for DMD Recruiting in Ohio: "Inclusion Criteria:

Males with Duchenne
Age 7 years or older
No signs of cardiomyopathy (preserved left ventricular systolic function)"

This study is taking place at two sites in Ohio: The Ohio State University (OSU) in Columbus and Cincinnati Children's Hospital Medical Center (CCHMC). 


Approximately 40 males with Duchenne will be enrolled in this study.

'via Blog this'

Summit Corporation PLC: Summit achieves SMT C1100 Phase 1 dosing milestone that triggers milestone payment

Summit Corporation PLC: Summit achieves SMT C1100 Phase 1 dosing milestone that triggers milestone payment: "SUMMIT ACHIEVES SMT C1100 PHASE 1 DOSING MILESTONE THAT TRIGGERS MILESTONE PAYMENT
* Trial moves to Multiple Dose stage
* Summit to Participate in Upcoming Industry Events
Summit (AIM: SUMM), a UK drug discovery company, today announced that it has successfully passed a milestone in the Phase 1 trial of SMT C1100 for the treatment of the fatal genetic disease Duchenne Muscular Dystrophy ('DMD'), which triggered the final payment from a $1.5 million funding agreement with US-based DMD organisations."

'via Blog this'

Friday 8 June 2012

Boys to Men Campaign in Scotland - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Boys to Men Campaign in Scotland - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: What a brilliant day at the Scottish Parliament. Over 30 family members, those living with Duchenne Muscular Dystrophy and supporters attended a lobby day at the Scottish Assembly on Wednesday 6th June to launch Action Duchenne's Boys to Men Campaign.

MSP's agreed to follow up with a debate in Parliament and Scottish families are arranging a meeting with Health Minister Michael Matheson MSP. 

Please get involved in our Boys to Men Campaign by writing to your MP and coming to Westminster on Wednesday 

All Sites Now Recruiting for GSK Exon 51 Skipping Trial

All Sites Now Recruiting for GSK Exon 51 Skipping Trial: The purpose of this study is to determine if GSK2402968 is effective in the treatment of ambulant boys with DMD resulting from a mutation thought to be corrected by exon 51 skipping. Two doses of GSK2402968 and placebo will be used in this study. This study is sponsored by GlaxoSmithKline (GSK).

This study will be conducted in the United States only and is now recruiting at 14 different sites in the following cities: Sacramento CA; Stanford, CA; Pensacola, FL; Iowa City, IA; Kansas City, KS; Baltimore, MD; Minneapolis, MN; St. Louis, MO; New York City, NY; Durham, NC; Cincinnati, OH; Columbus, OH; Portland, OR and Dallas TX.

Tuesday 5 June 2012

Webinar: New approaches to treating inflammation in Duchenne - PPMD Community

Webinar: New approaches to treating inflammation in Duchenne - PPMD Community: Parent Project Muscular Dystrophy�welcomes Dr. Michael Jirousek from�Catabasis�as our guest for a webinar on June 6, 2012 at 1pm eastern.*

Topic:�New approaches to treating inflammation in Duchenne
When:�Wednesday,�June 6, 1pm eastern

Inflammation is a key pathway in the disease pathway in Duchenne’s muscular dystrophy. CAT-1004 is a novel, anti-inflammatory agent being developed by Catabasis as a potential treatment for patients with Duchenne. This presentation will describe the science and rationale behind the development program.

Friday 1 June 2012

Summit Corporation PLC : Summit Announces Initiation of Phase 1 Trial for the Treatment of Duchenne Muscular Dystrophy

Summit Corporation PLC : Summit Announces Initiation of Phase 1 Trial for the Treatment of Duchenne Muscular Dystrophy: OXFORD, UNITED KINGDOM--(Marketwire - May 25, 2012) - Summit (AIM: SUMM), a UK drug discovery company, today announced that it has dosed the first cohort of patients in a Phase 1 study of SMT C1100 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal, rare genetic disease characterized by rapidly worsening muscle weakness. SMT C1100, an oral small molecule compound, is a potential disease-modifying drug that works to increase, or upregulate, the amount of a naturally occurring protein called utrophin.