Secretary Kennedy Adds MLD to Nationwide Newborn Screening

It took me a moment to come down from the momentum, adrenaline, shock, relief, celebration and joy that came from the last month of 2025. The hopes/wishes I expressed in my last post turned into a reality. On Tuesday, December 16, 2025, Secretary Robert F. Kennedy Jr. added both MLD (Metachromatic Leukodystrophy) and DMD (Duchenne Muscular Dystrophy) to the RUSP (Recommended Uniform Screening Panel) – AKA the foot prick blood test that every baby gets after they’re born.

And it happened much sooner than I expected.

Following our most recent visit to D.C. in the beginning of December (where Keira and I had a meeting at the White House and spoke on a panel at a Congressional Briefing), just a few days later we were invited back for an intimate meeting with Secretary Kennedy. Little did we know, this meeting ended up being a ceremony that was live-streamed and one hour before it started I was told that they would like me to say a few words.

As we walked into the “meeting” room in the Hubert H. Humphrey Building on Independence Avenue, we were surprised to see a U-shaped seating panel in front of many flags with two TV screens on either side. A giant camera was in the middle to live-stream the occurrence and behind it were rows of seats. We assumed these rows were for us, the MLD families in attendance. A few seats had names on them but none for us. As we settled in, Dave turned to me and said “I think your seat is actually up there…” pointing to the U-shaped panel. Shockingly, he was right, it was Secretary Kennedy seated next to his Chief of Staff Dr. Stephanie Haridopolos and to her left was myself, followed by two other parents. To the Secretary’s right were a number of Governors who had supported the reason we were all there that day: to add these two rare diseases who have known curative treatments to nationwide newborn screening.

I didn’t know until that morning what exactly was going to happen at this meeting. And I was in tears when we found out. It was hard not to tear up again as it came time for me to speak, but thankfully I held it together and kept my remarks short and sweet. As I mentioned in my remarks, it was an extremely bittersweet moment with Dave, myself, Eva and Keira in attendance while Livvy was at home with her Grandma, unable to walk, talk or benefit from the treatment her sister received 5 years ago.

But thanks to Secretary Kennedy, his team at HHS, the Governor’s in attendance, as well as those who helped support these efforts along the way – including AZ Congressman David Schweikert and Representative Greg Stanton – soon, no other family will have to live through what we have: losing one child in order to save another.

It truly is a miracle that just over 5 years had gone by since the girls were diagnosed, and in that time the treatment Keira received became FDA approved and now it’s been added to recommended newborn screening panels for the United States. The many families before us have truly paved the way, and worked for decades to get to this point. I sincerely hope the speed with which the treatment became FDA approved and the time until screening is recommended continues to lessen. For MLD alone, one baby is born with it each week. So the longer we wait to implement newborn screening, the more babies will face the same fate as our daughter Livvy (an average lifespan of 6 years old).

And while this addition is a monumental step forward for families like ours, our work isn’t done. Now, the 15 states that are RUSP-aligned (AZ among them), have to actually set up their labs to include the test for MLD. Which can take up for 18-24 months.

Since we returned from D.C. on December 17th, I’ve already emailed both Congressman Schweikert and Representative Stanton’s office, sharing Secretary Kennedy’s actions to include MLD and DMD, along with why it’s so important to implement testing sooner rather than later. I hope that the state of Arizona and its newborn screening department realize the gravity of this situation and how many lives they will save by acting swiftly.

See the full live stream of the ceremony here:

See below for a post from HHS from this meeting, that includes a group photo along with a shot of Secretary Kennedy giving Keira one of his challenge coins.

And below is an article from our local newspaper, the Ahwatukee Foothills News, recapping our family’s journey to this point.

Congressional Briefing #2

While our trip to D.C. in May was both successful and hopeful in sharing our family’s story with lawmakers, truly moving the needle seemed to warrant another trip and another Congressional Briefing.

This past week, Keira and I returned to the Capital, where we were included in a panel discussion about our family’s experience and how newborn screening for MLD could change – and save – the lives of so many.

Included among the panel were the following:

It was a great discussion with perspectives from each of the panelists.

One glaring statistic I learned: the test for MLD newborn screening (to be added to the foot prick test after birth) only costs $5-8 per baby! Compared to the millions in costs for a single symptomatic child, like our daughter Livvy, who will need a variety of special needs equipment, 10+ medications/day, and numerous hospital and ER visits (to name a few things) before passing away at an average age of 6.

In attendance were other MLD families as well as members of a variety of House and Senate committees. The feedback seemed very positive.

Earlier that day, we also had an unexpected meeting with the White House Domestic Policy Council staff to similarly share our family’s story and the importance of adding MLD to newborn screening when there has been an FDA approved treatment for it for well over a year and a half.

It was also a positive meeting, and little did we know it may have actually made a difference.

A mere 48 hours later we were invited back.

I don’t know what this meeting entails, but we are VERY hopeful that it will include ensuring nationwide screening for MLD so that any child diagnosed will have access to the same FDA approved treatment that Keira received.

We will keep everyone updated!

In the meantime, here are a few photos from last week’s trip to D.C.

Recap of MLD on the Hill

In May of this year, we had the incredible opportunity to go to Washington D.C. with other MLD families, doctors, scientists and supporters to share our stories, and why Newborn Screening is necessary for MLD.

Time got away from me so I’m just now posting about it…one week before we return to D.C. (more on that later).

During our initial trip, where I took Keira and Eva and Dave stayed home with Livvy, we met with several lawmakers (and their teams) and I was among a few parents who spoke at a Congressional Briefing on the Hill. Not something I had on my bingo card for 2025…or ever, for that matter!

It was an amazing experience but also very eye opening to see in-person the inner workings of our government and the processes by which things get done (or sometimes don’t). Not unlike having to be an advocate for your children at the hospital, it is up to us at citizens to be an advocate for things we know are important to this nation – and in this case, are life-saving.

Following the ACHDNC being shuttered by the Trump administration – a mere one week prior to the vote on adding MLD to the RUSP – the entire MLD community was up in arms and determined to somehow move the vote forward.

So we, along with other MLD families across the nation, went to Capitol Hill to show lawmakers what newborn screening for MLD can do; to show them, in person, the lives that have already been saved.

During the trip, we met with Rep. Greg Stanton, along with staffers from the offices of Senator Mark Kelly, Senator Ruben Gallego and Rep. Buddy Carter from Georgia.

I also spoke alongside three other parents at a Congressional Briefing at the Capitol Hill Visitor Center, where a variety of lawmakers listened to our stories of tragedy and how they have the power to turn this into a triumph for the many families who will also be faced with this devastating diagnosis.

Below are a few photos from our trip. Keira and I are looking forward to going back to DC next week! Feel free to follow along on that journey on my Instagram, @mrskendrariley.

Kendra Riley and her daughters Eva and Keira meet US Representative from Arizona, Greg Stanton at his office in the US Capitol in Washington, DC on May 7, 2025 to advocate for gene therapy and newborn screening for MLD.

Why We Need Newborn Screening for MLD

Now that the U.S. has an FDA approved treatment for Metachromatic Leukodystrophy (MLD), you’d think they would automatically add it to the nationwide Newborn Screening recommendations for all states in order to immediately begin saving lives, right? Wrong.

Accomplishing this is also quite the process and one that requires the entire MLD community to share their stories, educate the committee who makes the decision as to what goes on the RUSP (Recommended Uniform Screening Panel) and make it completely obvious that doing so would save thousands of lives (especially now that there is an approved treatment which must be given before children become symptomatic in order for it to be effective).

So, who is this committee? It’s called the ACHDNC, or Advisory Committee on Heritable Disorders in Newborns and Children. According to their website, it is comprised of 10+ doctors whose specialties range from pathology, genetics and public health practice to laboratory services, pediatrics and molecular biology (to name a few). It is they who determine whether a disease gets added to the RUSP and therefore recommended to every state in the nation to be included in the foot prick blood spot test that every baby gets at their first visit to the pediatrician.

Only then will MLD be diagnosed in time for a child to receive the FDA approved treatment. Without it, babies with MLD will ultimately become symptomatic, and it will be too late. But they may be able to spare their future children now knowing it’s genetic. Just as we have with Livvy and Keira, families across the nation will be forced to lose one child in order to save another. Which is absolutely unacceptable when there is this life-saving treatment available in the U.S.

So, what do we as a community do? We speak up.

Last year, myself along with many other MLD families made public comments to the ACHDNC in support of adding MLD to the RUSP.

You can view my remarks (with Livvy by my side) below at the 1:33:52 mark.

Today, on Friday, May 10th, I’ll be speaking with the committee again. But this time, I’ll have Keira with me so they can see for themselves the power of this gene therapy treatment to not just transform but save lives.

To think this therapy has been approved since March 18, 2024…just imagine how many babies have been born since then whose parents have no idea their child could become symptomatic with MLD and by the time they find out it will be too late.

That is exactly what we are trying to prevent. Hopefully they hear us loud and clear.

FDA Approves Gene Therapy Treatment for MLD

It’s been a while since I’ve posted to the blog, but not for lack of updates…lack of time.

While much has happened since my last post in November 2023 (Keira turned 4, I continued advocacy efforts for Right to Try 2.0, which you can read in the Wall Street Journal, USA Today and The Epoch Times and all three girls immediately became Swiftiies after seeing the Eras Tour on TV). But one of the most important updates is that the FDA approved the gene therapy treatment Keira received in Italy (called Libmeldy in Europe and Lenmeldy in the US)!

If you haven’t yet read the details of the FDA approval, you can find it here.

We have been overwhelmed with emotion, thinking of all the families who will be spared from losing a child to this disease now that this treatment is approved. A huge round of applause goes to all of the doctors, nurses, advocates, gene therapy families, and many more who have had a role to play in this process. It has been years in the making!

Since the approval, I have also had the honor of sharing the girls’ story with CNN and NewsNation, which you can watch here:

It’s always bittersweet to share the girls’ story with the world, but I know it’s an important one that is helping to create massize change for other families like ours.

Thank you to all of you have been along with us on this journey!

Next step: getting MLD added to nationwide Newborn Screening so that all babies are tested in time to actually get the FDA approved treatment! More on that soon…

Advocacy in Atlanta and London

This month has been a whirlwind of travel – first to Atlanta to speak about Right to Try 2.0 at the State Policy Network’s 30th Annual Conference and then to London to speak about the gene therapy Keira received, how it has transformed her life and how we are helping other families like ours. Next? To Milan for Keira’s 2-Year post- gene therapy check up! Time flies!!

I was so honored to be included in these opportunities this month and I hope it only sheds a light on the legislation that needs to change to help families and patients like our daughters.

I was shocked – in a good way – to see Keira’s sweet face on every seat in the room when I walked in to speak. The face of hope for other patients striving to get that one treatment in the world that could change their life right here at home in the US, as opposed to having to raise money and uproot their lives for access to life-saving treatments.

I am so grateful for the Goldwater Institute who invited me to SPN’s 30th Annual conference to speak at their panel on “How Your State Can Unleash the Future of Medicine”. They are an amazing organization doing so much across the nation to make positive change. My time with them only inspired me to do more.

From Atlanta, I headed across the pond to London to speak to the staff of the genetics company who was instrumental in treating Keira.

Their 170+ staff rarely get to actually see the results of their hard work so I was amazed at the feedback I received sharing how well Keira is doing today and the work our family has done to help others.

They are true miracle workers and it was an unbelievable honor to meet them and share that it’s because of them we get to see miracles on a daily basis through Keira.

Next stop: Milan, Italy! 🇮🇹

PFDD for MLD on Oct 21st

These letters may not mean much to you but they mean SO much to the MLD community of patients and their loved ones. Because on October 21st, a select group of individuals in that community (including yours truly), get to share their stories in a PFDD, meaning Patient Focused Drug-Development meeting, with the FDA, researchers, clinicians, and other stakeholders. Yes…I said the FDA!

I am blown away that we get to shine a light on this horrible disease and share that there is actually a treatment option out there that can save non-symptomatic patients with this disease, like Keira.

And YOU can be a part of it! Both the speakers, as well as their family and friends, can log on to this Zoom discussion to share what you see first-hand regarding this disease and how a treatment like the gene therapy Keira received could be beneficial (which, by beneficial, I mean the absolute best option on Earth).

Myself and other panelists meeting on Zoom to practice our presentations for the Oct 21st PFDD.

Here is the link to register and join us on Zoom on October 21st: www.mldpfdd.org.

This PFDD means quite a few – very important – things:

1. Our community has raised enough awareness about this terrible disease to get in front of the FDA, researchers and other key stakeholders and share its affect on patients and caregivers and the only treatment options that exist.

2. This could raise awareness for the need for the Newborn Screening panel to include MLD. Because the gene therapy Keira received has come to the US (and is already approved in the EU), we have a way to treat newborns diagnosed with MLD. And unless it’s treated fast, they will end up with a fate like Livvy’s.

3. Once they see the odds this community is up against and the options available, they may be more open to approving treatments for life-ending disease. No child should die by age 6 when they have the chance to live a full life if they were diagnosed at birth.

As part of the panels presenting, I will am getting to share our family’s story in relation to current and future treatments for MLD. Including, the two treatment options we used for the girls (Livvy’s clinical trial and Keira’s gene therapy), any complications that resulted (like Livvy’s internal port in the clinical trial), how we manage Livvy’s symptoms, how Keira is symptom-free nd how willing we are to do anything to give our children the chance at a better life.

Here is the link again to stay up to date and join us online October 21st: www.mldpfdd.org. By registering you will also have access to the September 12th community webinar.

If you or a family member have been affected by this disease I highly urge you to join me – attendees can share their insights too!

UPDATE: to see the full MLD PFDD, including my testimony, it is now online here: www.mldpfdd.org.