NEWPORT BEACH, Calif., April 5, 2017 – CureDuchenne, the leading nonprofit focused on finding a cure for Duchenne muscular dystrophy, is proud to sponsor SB 643 (Pan) which passed the Senate Health Committee today with a vote of 8-0. SB 643 will add Duchenne to the Genetically Handicapped Persons Program’s (GHPP) list of eligible medical conditions. A coalition of rare disease organizations, Duchenne organizations, parents, patients, hospitals, health associations and healthcare providers supported SB 643.
Duchenne muscular dystrophy is a fatal genetic disorder that causes muscle degeneration and typically effects boys. Currently there are nearly 15,000 boys in the U.S. battling this disease and 300,000 worldwide. Most boys with Duchenne lose their ability to walk by age 12 and do not survive beyond their mid-20s. As the disease progresses, the simplest of tasks become difficult, and in the later stages, heart and breathing muscles begin to fail. At this time, there is no cure for Duchenne.
Patients with Duchenne in California currently receive specialized coordinated care through the California Children Services program (CCS), a state program that serves children with rare, complex health care needs. When they age out of the CCS system at 21 years of age, their families are left to navigate a healthcare system that provides very little specialized care and support.
The GHPP is a state health care program for adults with certain genetic diseases. The GHPP provides complete coordinated services to its clients by working closely with doctors, nurses, pharmacists, and other members of the health care team. When the GHPP was created, the mean age at death for patients with Duchenne was around 19 years. Fortunately, various interventions which are available through specialized neuromuscular care centers, have led to improvements in quality of life and longevity so that children who are diagnosed today have the possibility of living longer. Duchenne is a multisystem disease that impacts a patient’s biological (neurology, cardiology, orthopedics, pulmonary, etc.) and mental health, so one aspect of care cannot be taken in isolation. Therefore, access to wraparound services like those that are offered through the GHPP are key features of treatment.
SB 643 supporters include CureDuchenne (sponsor), Braedan’s Bridge; Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine at UCLA; California Children’s Hospital Association; California Medical Association; Children’s Specialty Care Coalition; Coalition Duchenne; Duchenne Therapy Network; EveryLife Foundation for Rare Diseases; Global Genes; Hope for Javier; Lucile Packard Children’s Hospital & Clinics; Muscular Dystrophy Association, National Organization for Rare Disorders; Parent Project Muscular Dystrophy; Stanford Health Care; and UC Davis Health.
CureDuchenne was founded in 2003 with a focus on saving the lives of those with Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. With support from CureDuchenne, nine research projects have advanced to human clinical trials. CureDuchenne also is the innovator bringing physical therapy and standard of care to local communities around the country through CureDuchenne Cares. For more information, please visit CureDuchenne.org and follow us on Facebook, Twitter, Instagram and YouTube.