Three pharmaceutical companies have partnered with CureDuchenne to support CureDuchenne Cares, a physical therapy, education and outreach program for the Duchenne muscular dystrophy community to help establish and provide consistent standard of care for Duchenne patients. BioMarin Pharmaceutical, PTC Therapeutics and Sarepta Therapeutics are supporting the program to provide a much needed resource for the Duchenne community.
CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, joined forces with Jennifer Wallace, physical therapist and founder of the Duchenne Therapy Network,to develop the program to help educate physical therapists, parents and caregivers on the best standard of care for Duchenne patients. The goal of CureDuchenne Cares is to improve outcomes, extend ambulation and provide guidance on standards of care for all Duchenne patients.
“Physical therapy and daily stretching is critical for the overall health of Duchenne patients,” said Dr. Brenda Wong, Director of the Comprehensive Neuromuscular Center at Cincinnati Children’s Hospital Medical Center and Professor of Pediatrics and Neurology at the University of Cincinnati. “Lower extremity contractures negatively impact motor functional activity for ambulation and stance. Physical therapy with supervision for a daily home stretching program with use of night resting ankle braces from early stage of Duchenne is needed to maintain range of motion in the lower extremities and optimize motor function.”
To increase the health and wellbeing of patients living with Duchenne, CureDuchenne Cares education and outreach includes best practices for Duchenne management, stretching, equipment, orthopedics, nutrition, and adapting all of these factors to the different stages of the disease. Duchenne is a progressive muscle-wasting disease that currently has no cure. Boys with Duchenne are usually diagnosed by 5, in a wheelchair by 12 and most don’t survive their mid-20s.
CureDuchenne Cares features educational sessions for parents and physical therapists; instructional videos; educational materials and blogs; and information on the latest scientific developments in Duchenne research, including status and participation in clinical trials. The CureDuchenne Cares team travels to local communities around the country to bring these resources to the patients and parents where they live and at Duchenne clinical trial sites. Upcoming CureDuchenne Cares sessions include:
- June 5, San Diego, CA– physical therapist continuing education course
- June 6, San Diego, CA– parent/caregiver workshop
- June 26, Long Beach, CA– physical therapist continuing education course
- June 27, Pasadena, CA– parent/caregiver workshop
- July 17, Maple Valley, WA– physical therapist continuing education course
- June 18, Maple Valley, WA– parent/caregiver workshop
Additional sessions will be scheduled throughout the rest of the year and will be announced soon. For more information about the CureDuchenne Cares sessions, please call 949-872-2552.
“Cure Duchenne definitely cares. We as a family felt that they have always offered great support, the knowledge to obtain the funding and support the clinical trials that we know are truly on the edge of a breakthrough,” said Tammy Henegar, Duchenne parent from Curtace, Ohio. “Having CureDuchenne Cares was huge for the Toledo area. We gained much knowledge and expertise from the session as parents. The physical therapists and occupational therapists were extremely happy with the session learning more about Duchenne and how to help the boys that are in their care in ways that they may not have known.”
“With support from BioMarin, PTC and Sarepta we are able to expand the CureDuchenne Cares program and fill an unmet need for the Duchenne community,” said Debra Miller, founder and CEO of CureDuchenne. “We need physical therapy that can help our sons walk longer, which will allow them potentially to benefit from new medicines when they become available. Unfortunately, some boys have been disqualified from clinical trials that could possibly improve their health because they did not have proper care or physical therapy. CureDuchenne Cares provides ongoing education and outreach for Duchenne families as we continue to fund research to find a cure for this devastating disease.”
CureDuchenne has funded seven research projects that have advanced to human clinical trials. CureDuchenne has leveraged $100 million in pharma and biotech research and development investments. Now three of the projects CureDuchenne supported with funding, Prosensa Holding N.V. (recently acquired by BioMarin), Sarepta Therapeutics and PTC Therapeutics, are the closest to becoming the first drugs to be approved for the treatment of the disorder.
CureDuchenne is a national nonprofit organization located in Newport Beach, Calif., dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys worldwide. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. With the help of CureDuchenne’s distinguished international panel of Scientific Advisors, funds raised by CureDuchenne support the most promising research aimed at treating and curing Duchenne. To date, seven CureDuchenne research projects have advanced into human clinical trials – a unique accomplishment as few health-related nonprofits have been as successful in being a catalyst for human clinical trials.
About Duchenne Therapy Network
Duchenne Therapy Network was created to help those who have been diagnosed with Duchenne Muscular Dystrophy find a physical therapist skilled in this specific area. It was founded by physical therapist Jennifer Wallace. Duchenne Therapy Network provides clients and families with excellent healthcare practice, individualized and comprehensive assessment, treatment and follow up within the scope of practice. Duchenne Therapy Network provides referrals to a network of healthcare providers to address areas of care for clients. Duchenne Therapy Network provides best practice standards, based on expert knowledge and research evidence to healthcare professionals on a regular, periodic schedule to ensure timely, accurate and standard delivery of information.