MISSION:
Dedicated to changing the predicted outcome for all boys with Duchenne muscular dystrophy
Transforming Care
Studies have proven that access to multidisciplinary care adds on average TEN YEARS to DMD boys’ life expectancy.
The care landscape has advanced dramatically in the past 35 years – but not all advances impact equally. Hope for Javier partnered with STONY BROOK CHILDREN’S HOSPITAL to change that by creating a comprehensive care model for this patient population. Stony Brook Children's Hospital is now a Certified Duchenne Care Center. The accreditation comes from Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne).
This accreditation makes Stony Brook the only certified center on Long Island and in the NYC metropolitan area; it is now one of only two centers in the whole of New York State.
This has brought equity in health outcomes, as well as access to clinic trials across economic, geographic, racial and ethnic groups.
Funding Scientific Research
The costs involved in developing a new drug are staggering.
Many promising discoveries never make it out of the lab, into the therapeutic development process because of a lack of funding. These potentially important discoveries cannot cross the ever-widening gap in funding and support for the type of research that moves basic science from the lab toward therapeutic development. This gap has come to be known as the “Valley of Death”.
That is where we come in; Hope for Javier provides bridge funding (fills a gap) that allows researchers to continue their work while waiting on or working towards larger grants from government agencies.