Right to Try

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! 🇮🇹

Right to Try 2.0 Passes in Arizona

After testifying at the Arizona Senate and House of Representatives, sharing our family’s story and how the expansion of the original Right to Try Bill could help families like ours, I’m thrilled to share that Right to Try 2.0 has passed and was signed by Governor Doug Ducey!

Right to Try 2.0 is a first-in-the-nation reform that will empower seriously ill Arizonans to access personalized treatments that have yet to receive full approval from the U.S. Food and Drug Administration.

Goldwater Institute

We had the pleasure of meeting Governor Ducey last week during the Ceremonial Bill Signing at the Arizona State Capitol. The girls were thrilled to go (after a quick lesson in US government so they knew what a Governor, Senator and a Bill is).

Governor Ducey, Senator Nancy Barto and myself all spoke before he signed the Bill. You can view a portion of that here:

The Goldwater Institute’s Right to Try for Individualized Treatments reform builds on the original Right to Try law and protects patients’ right to try to save their own lives by seeking treatments tailor-made for them that are not yet FDA-approved. This common-sense measure accounts for new innovations in medicine and helps get those innovations to the patients who need them the most.

As I mentioned in my speech, the United States is the greatest country on earth and access to life-saving medical treatments should be among its many benefits. We are thrilled Arizonans now have that right and are hopeful other states – and eventually the nation – will follow suit.

You can read more about Right to Try 2.0 and our efforts here on Reason.com.

And here some photos from the signing at the Arizona State Capitol:

Photos by Neal Mei Photography, courtesy of the Goldwater Institute.