MLD Foundation

07/14/2026

Looking for any researchers who would like to explore if MLD carrier status causes older carriers to exhibit neurological problems. Please contact MLD Foundation.

The Premier Family Resource for Metachromatic Leukodystrophy Information & Support since 1999!

07/06/2026

Congratulations to our friends at Cure MSD! This is a huge moment for them!

A significant milestone for the rare disease community! The U.S. Food and Drug Administration has cleared the Investigational New Drug application for an investigational gene therapy designed to treat children with multiple sulfatase deficiency (MSD), an ultra-rare, life-limiting genetic disorder.

This first-in-human study represents years of scientific research and collaboration across the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC), bringing together patients, families, advocates, clinicians, researchers, and consortium partners to help advance this program to the clinic. Through coordinated manufacturing, clinical and regulatory efforts, the partnership helped pave the way for this important step forward.

Read more about the collaborative effort to accelerate potential new treatment options for children and families affected by MSD: https://fnih.org/news/bgtc-msd-ind-clearance/

07/02/2026

I didn’t know July was Bereaved Parent Month. This post from Bereaved Parents of the USA spoke to me and holds a sentiment that I believe is shared by parents who have lost a child, and those in the MLD community who have lived through their children gaining their butterfly wings. Sending hugs to all!❤️

🦋🦋🦋🦋🦋🦋🦋🦋🦋🦋🦋🦋🦋🦋🦋🦋

Today begins Bereaved Parents Month.

For most people, July is just another page on the calendar.

For us, it is a month that quietly says, “We see you.”

There are millions of parents who have buried a child.

You pass us in the grocery store.
You sit beside us at work.
You wave to us at the ball field.
You laugh with us at restaurants.

Many days, you would never know.

Because bereaved parents become experts at carrying the unbearable while looking like we’re carrying nothing at all.

This month isn’t about asking for sympathy.

It’s about recognizing a love that didn’t end when a heartbeat did.

It’s about remembering that every accomplishment, every holiday, every family photo, every birthday, every ordinary Tuesday still has someone missing from it.

It is about speaking our children’s names without lowering our voices.

It is about acknowledging that we are still parents.

Always.

Bereaved Parents Month is a reminder that our children are not forgotten. It is a time to recognize the parents who continue to love, miss, and carry their children every single day. It is an opportunity for others to pause, remember, and better understand a journey that never truly ends.

If you are a bereaved parent, we hope you’ll see yourself here.

If you love someone whose child has died, stay with us this month. Listen. Learn. Remember with us.

Because our children mattered.

They still matter.

And so do the parents who carry them in their hearts every single day. ❤️

06/22/2026

If you, family or friends are in Northern NY, specifically Hermon, this coming Sunday, consider joining the fun at the annual Walking for Adelyn event in remember of Adelyn Wayerling, a little girl who passed away a year ago from MLD. Proceeds benefit MLD Foundation.

Check it out! https://mld.foundation/walking-for-adelyn/

05/27/2026

Oregon NBS Advisory Board
Votes to Move MLD
onto Step 2!

05/12/2026

The past week and a half has been filled with what MLD Foundation was founded to do 25 years ago!

Meeting, supporting and being a resource for families is the reason the foundation originally came into being. We had the pleasure and privilege to meet a mom and her daughter in Tallinn, Estonia and spend the day with them on our way to the MLD NBS Alliance meeting in Prague. The daughter, Linda, reminded us of our own daughter, Lindy, when she was first diagnosed. To be able to connect and talk about the road ahead meant everything! The MLD journey is isolating and hard enough but it’s even more isolating and harder when you believe you are the only case in a country. Making a personal connection can bring hope and a feeling of community. Through out the 25 years of the foundation we have been able to do this when we travel to other countries as well as throughout the U.S. It’s meant the world to those we have met.

I had another opportunity to meet and hear the story of a lovely MLD family in Prague at the 8th MLD NBS Alliance put on by Orchard Therapeutics. This family’s two children received the Arsa-cel gene therapy. They too are the only known MLD family in their country of Czechia. Being able to connect with them and to not only be a resource but also a connection to others in the MLD community is heartwarming and hope giving.

At the start of our trip, while waiting to board our plane, Dean was able to give invited public comment during the second committee meeting to add MLD NBS to the Wisconsin state panel.

Taking part and sharing at the 8th MLD NBS Alliance meeting was filled with rich discussions networking, and information about past, present and future research and MLD NBS internationally.

The end of our travel was capped off by a visit with Alex Kemper at Nationwide Children’s Hospital in Columbus, OH who lead the ACHDNC’s external expert review of the MLD NBS RUSP nomination. We explored future collaborations, geno/pheno type correlations, consent and ethics on this day celebrating the 25th year of MLD Foundation’s legal formation.

This is just a glimpse of what occurs behind the scenes of MLD Foundation.

Those desiring to donate to MLD Foundation may do so at https://mld.foundation/donate

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/