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.

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'

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.

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.

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.