MLD FOUNDATION
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Mission Statement
We C.A.R.E.® ... Compassion for families, increasing Awareness, influencing & funding Research, and promoting Education for metachromatic leukodystrophy, a very rare terminal genetic neuro-metabolic lysosomal disease where over half the cases affect infants.
About This Cause
Formed in 2001, MLD Foundation has been serving the MLD community around the globe for well over 20 years. With a gene therapy approved in the EU and under FDA review (Q1'24 PDUFA reporting date), identifying newborns with MLD and getting them to therapy as soon as possible will minimize progression and maximize the outcomes. We've been working on MLD newborn for over a decade and will be submitting a RUSP nomination for federal review in Q1'24, followed by extensive work to implement MLD NBS in every US state. Access and Reimbursement is also critical - cross state line insurance and Medicaid coverage for a multi-million dollar "one and done" therapy is not trivial to put in place - we've been working on policy and practical issues to resolve these issues for many years.