CureDuchenne has announced its enthusiastic support and sponsorship of California Senate bill SB 643, which adds Duchenne muscular dystrophy to California’s Genetically Handicapped Persons Program (GHPP) list of eligible medical conditions.
The bill, introduced Feb. 17 by Richard Pan (D) — a pediatrician and state senator representing the Sacramento region — passed the California Senate Health Committee on April 5 by an 8-0 vote. The legislation is supported by a diverse coalition of rare disease and DMD organizations, individual patients and parents, hospitals, health associations and other healthcare providers.
DMD is a rare genetic neuromuscular disorder characterized by progressive symmetric muscle weakness and degeneration associated with progressive loss of contractile function. According to CureDuchenne, an estimated 300,000 boys and young men have DMD worldwide, including nearly 15,000 in the United States. Most boys with Duchenne lose the ability to walk by age 12 and rarely survive beyond their mid-20s. Even the simplest day-to-day tasks become increasingly difficult as the incurable disease progresses.
In California, Duchenne boys receive special coordinated care required by California Children Services (CCS), which assists children with rare, complex healthcare needs. But when these individuals turn 21 — an age at which patients with DMD typically become severely disabled — they also become ineligible for CCS support, leaving them and their families with few options.
The GHPP, a statewide healthcare program for adults covering a specific list of genetic diseases, provides its clients with comprehensive coordinated services in collaboration with doctors, nurses, pharmacists and other professionals. When lawmakers established the GHPP in 1975, the mean age for Duchenne patients at death was about 19 years. Since then, however, life expectancy for DMD patients has improved, and some boys diagnosed with the disease today may live well into their 20s and even beyond.
Because DMD attacks multiple systems in a patient’s body and affects mental health as well, no single aspect of care can be implemented in isolation, which makes access to targeted services like those offered through the GHPP key elements of DMD treatment.
SB 643 supporters include CureDuchenne, which is based in Newport Beach, Calif., and is also a sponsor. Others are Braedan’s Bridge; Center for Duchenne Muscular Dystrophy; UCLA’s David Geffen School of Medicine; the California Children’s Hospital Association; the California Medical Association; the Children’s Specialty Care Coalition; Coalition Duchenne; the Duchenne Therapy Network; the EveryLife Foundation for Rare Diseases; Global Genes; Hope for Javier; Lucile Packard Children’s Hospital & Clinics; the Muscular Dystrophy Association, the National Organization for Rare Disorders; Parent Project Muscular Dystrophy; Stanford Health Care; and UC Davis Health.