MLD Foundation

02/28/2026

Today we celebrate Rare Disease Day and the accomplishments that have been made so far. There is still much to do but today we celebrate the WINS!❤️

MLD Foundation is thankful to HHS Sec Kennedy for adding MLD NBS to the RUSP and to Stephanie Haridopolos, Chief of Staff/Senior Advisor to the Surgeon General, for being our champion and the whole MLD community working together to accomplish this goal!

02/12/2026

This winter has been so hard on the MLD kiddos. So many requiring hospitalization and others earning their butterfly wings so close together.

Sending love to all!

02/03/2026

This great news for all the rare disease community!

‼️Breaking News: Rare Pediatric Disease PRV Program Reauthorized by Congress!

After a two-year campaign to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, the rare disease community’s relentless advocacy has paid off.

Congress has passed the Labor, HHS, and Related Agencies Appropriations bill, effectively reauthorizing the PRV Program for five years while also funding a number of other critical healthcare agencies.

We applaud the reauthorization of the PRV Program and renewed investments in critical health research and public health programs.

Thank you to the congressional champions who have partnered with our rare disease community to secure these advances. While significant work remains to enable all those living with rare diseases to thrive, today’s progress will accelerate innovation, expand access to life-changing therapies, and offer renewed hope to children and families whose futures once seemed beyond reach.

To learn more about the full healthcare package, please visit our website: https://everylifefoundation.org/congress-passes-five-year-reauthorization-of-rare-pediatric-disease-prv-program/

02/03/2026

MLD Foundation wants to wish Michael Gelb super congratulations for being awarded the Catalyst Award at the 22nd WORLDSymposia in San Diego. It was way over due. Professor Gelb has done so much for the rare disease community and especially for MLD by developing the phenomenal MLD NBS which is now on the RUSP (the recommended uniform screening panel) and will be saving babies and changing their lives as they receive Lenmeldy gene therapy! So honored to work with him and call him our friend!

12/30/2025

As 2025 comes to an end and we usher in 2026, MLD Foundation wants to wish our MLD family, their families, and friends a Happy New Year! We celebrated a momentous event when MLD NBS was added to the RUSP but there is more to do. Your year end, tax deductible gift to MLD Foundation helps ensures we will be there to support MLD families, MLD NBS will be on every state NBS panel, and the research for life changing therapies continues. May 2026 be another year of celebrations for the MLD community!
MLD.foundation/donate

12/23/2025

12/22/2025

If you’re looking for a last minute gift, this is a great read! The main character’s child has MLD and depicts the MLD journey well even though the author hasn’t experienced MLD personally. Don’t miss how you can donate to help MLD Foundation continue its work under the author’s note.
Happy Holidays!
Order off Amazon

https://a.co/d/4OGbnJR

If you are on Amazon and search for Caleb Stephens books the audio version will come up but if you tab on the app dio version it will allow you to choose paperback.

12/17/2025

It finally happened, after many years of work, tears, overcoming barriers and hurdles by many - MLD NBS is on the RUSP! While it’s bittersweet for those who have endured this disease, there is now hope for the future!