SB 643 Passes Senate Health Committee

The First Step to Add Duchenne Muscular Dystrophy to GHPP

NEWPORT BEACH, Calif.–(BUSINESS WIRE)–#DuchenneCureDuchenne,
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.

About CureDuchenne

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.

Contacts

CureDuchenne
Karen Harley, 949-872-2552