Giving Tuesday is not just about today

MLD Foundation is small but mighty – powerful, connected, influential, and active! As you consider your Giving Tuesday gifts today, please think about us and know the importance of every gift, no matter how large or small.

As we look into the near future, this is what your support is enabling us to do:

• Most importantly – being a hands on resource and support for families with MLD … whatever it take, whenever it is need, and no matter what form or time of day that support takes … direct personal loving and ongoing contact.
• Resuming our in person MLD Family Conference™ in 2023!
• Working with the FDA and Orchard Therapeutics to help get their gene therapy into review and approved in the USA – just like we did in Europe and the UK!
• Provide global support, education, and referrals for newly diagnosed families, many of whom need support for a formal diagnostic confirmation and because of progression are not eligible for therapies so they need clinical and encouraging support.
• Managing the MLD Newborn Screening RUSP Nomination Expert Advisory Group and US MLD NBS nomination process to open the door to screening across the US and internationally. We’re writing the Nomination and will present it to the ACHDNC in 2023.
• Improving the correlation of MLD genetic sequencing to predicting the form of disease to improve newborn screening therapeutic referrals and clinical care.
• Writing, supporting, educating and in person state and federal lobbying for policies that benefit rare disease (including MLD) families and their loved ones – we’re also active members of numerous collaborative efforts on policy. Current focus includes better MEDICAID and CMS policies, coverage and payments for emerging multi-million dollar gene therapies, newborn screening, appropriations (government funding) for newborn screening and better clinical care, and overall reimbursement and pricing policies.
• Working with more than a half dozen BioPharma’s on next generation MLD therapies targeting differing therapeutic strategies and forms of MLD, including the two current active MLD clinical trials, and investors in emerging companies and therapies.