Mila's Miracle Foundation to Stop Batten

03/27/2026

After a year of hard work, it's very exciting to see Critical Path Institute officially launch the 'One to Millions' initiative to support the near future where patients receive individualized medicines at scale.

The U.S. Food and Drug Administration and Medicines & Healthcare products Regulatory Agency are shifting toward Process Approval, but once we enter this new world, setting Standards based on publicly shared learnings and data across dozens and eventually hundreds of medicines will be fundamental. C-Path offers just what we need right now - the expertise in building databases, the central and public position, and the close relationship with the FDA, industry, academics and key groups around the world.

Another great step toward individualized medicines becoming routine!

BioCentury Article: https://www.biocentury.com/article/658923 (Non-subscribers can download it) - thanks to Steve Usdin!

03/10/2026

With political divisions driving people apart in the US and around the world, it's been powerful to see people of all walks of life coming together to change our outdated system of access to medicines to help millions of sick and dying children.

This is no longer a technology problem, it's a human problem.

The moral imperative burns strong, cutting across political parties and country lines.

I was honored to be a voice at U.S. Department of Health and Human Services (HHS) alongside friend and fellow Batten mom Judy Stecker, as we both fight for her son Wheeler and the millions who follow.

03/03/2026

This year marks 10 years since Mila was diagnosed with Batten disease and my life took a turn that has forever shaped me. Since then, I've been on a mission to save my daughter, and when it wasn't in time for her, to save the millions who follow.

We're living in both the most devastating time - with tens of millions of children dying of genetic diseases - and the most promising time when science is no longer the barrier and existing therapeutic technologies can save the lives of thousands and soon millions.

But connecting the masses in desperate need with life-saving science means changing a system - one that has been in place for more than 50 years and still moves one medicine for one disease at a time. Unfortunately, this system simply doesn't work for patients with genetic diseases, nearly all of which fall under 10,000 small or rare conditions.

In my 10 year fight, I've learned that changing the system has been anything but linear. Three steps forward, one step back.

Last week, U.S. Department of Health and Human Services (HHS) and the U.S. Food and Drug Administration (FDA) rolled out the new Plausible Mechanism pathway. The guidance is the first to use the words "for Industry" associated with individualized medicines, acknowledging that these can no longer be one-off Hail Mary's if we want this approach to become routine, but instead must be approved medicines with an expectation for reimbursement. While there is still much work to be done, this is a big step toward Process Aproval - approving the WAY we make medicines instead of one medicine at a time. It's a logical, common sense solution that regulators around the world are aligning on, and it's the beginning of the commercially viable pathway I've been fighting for - the one that gets the flywheel going and moves us from Mila to Millions.

Onward!

VIDEO, HHS Roll-out: https://www.youtube.com/live/XWTO5khjaTQ?si=ZT0o_AneWN0bvowC



Tim Yu Judy Stecker Lowell Schiller Janet Woodcock Casey McPherson Allyson Berent Yiwei She Christina M. Hartman Winston Yan Fyodor Urnov David R. Liu Grace G. Amy Comstock Rick Tracy Beth Høeg, MD, PhD W***y Chertman Lawrence Tallon Julian Beach

02/11/2026

Today marks 5 years from when Mila died. I sit on her big bean bag chair and feel the emptiness in our home. I have no daughter to wrap my arms around, no cheek to kiss. The bright light of warmth she radiated through every room in our house isn't there any more. I sit and hold this poem a friend wrote for me, the English adaptation of her original in Chinese.

Today, on this February day, I will turn to the forest as I always do where I can feel Mila zipping through the trees, on the winds and in the birds. I find her in the little things.

02/06/2026

Quincy's parents live with a perpetual sense of guilt. If not for Quincy, it's for her sisters. If not for her sisters, it's for Quincy. They briefly consider outings or vacations with just Quincy's sisters for an occasional sense of normalcy, but the thought of doing anything fun without her seems impossible and weighs too heavily on their hearts. If Quincy is at home with a sitter, they know she is loved and happy, but the guilt is just too much. So her mom grabs those short windows when Quincy is napping or occupied to take her other girls out to get their nails painted so their life can feel relatively normal for just one moment.

This Is Today: https://www.this-is-today.com/berent-weisse-family

Featured Family: Allyson Berent .weisse Quincy's Quest Foundation Foundation for Angelman Syndrome Therapeutics

Photo Credit: Bonnekamp

02/04/2026

Quincy's parents' biggest fear is that if she lives past their lifetimes, who will advocate for her like they do? They are her cheerleaders on the field day and night, every week and month of the year. They know no one else could, or in the case of her sisters, should have to give her that same level of support.

To the world, Quincy is often a smiling, happy girl. But what many don't see is what her family does to ensure that she's the happiest she can possibly be - a sacrifice of what might be defined as a typical life that they choose to give up for her. In many ways, the struggle is not really Quincy's - it's her mom's, her dad's, and her sisters'.

There is beauty that comes with caring and protecting someone so deeply, but that beauty comes with a parent's greatest fear of when that is no longer possible.

This Is Today: https://www.this-is-today.com/berent-weisse-family

Featured Family: Allyson Berent .weisse Quincy's Quest Foundation Foundation for Angelman Syndrome Therapeutics

Photo Credit: Bonnekamp

01/30/2026

For Quincy, it's the smallest, yet most magical, things in life that bring her immense joy. It's the magic of a bubble - the shine, the glimmer, the moisture, the uniqueness of each one - if she touches it, it pops, then more come. She only has a few words, but one of her first was "bubbles". There aren't many activities that she and her sisters can equally enjoy, as their interests are harder to align with hers as times goes on. But playing in the water with bubbles never gets old for any of them - seeing bubbles, touching bubbles, being surrounded by bubbles.

That's what happiness looks like for Quincy, and in turn for her family. It's the magic of the small things that grounds them.

This is Today: https://www.this-is-today.com/berent-weisse-family

Featured Family: .berent .weisse Quincy's Quest Foundation Foundation for Angelman Syndrome Therapeutics

Photo Credit: Bonnekamp

01/27/2026

Quincy is 11 years old and has a genetic condition called Angelman syndrome that robs her of normal development, making speech, movement and learning an every day and every minute challenge.

Quincy's only real friends are her sisters. She speaks with her eyes and connects through touch, but when she's pushed to the limit of frustration, and doesn't have the words to express herself, she may pull their hair. She tries to make friends at school or on a playground, starting with soft noises and giggles, often staring at them for a response or even reaching to them for that touch and connection. But kids often don't understand and lose patience, and so they walk away.

This Is Today: https://www.this-is-today.com/photo-journal

Featured Family: .berent .weisse Quincy's Quest Foundation Foundation for Angelman Syndrome Therapeutics

Photo Credit: .Bonnekamp

01/21/2026

To kick off an already pivotal and exciting year, I decided to turn to the next chapter in my fight for children like Mila and accepted the CEO position at EveryONE Medicines (EOM), the company Tim Yu and I co-founded to develop and deliver individualized medicines to patients at scale.

I'm wearing a new hat, but my mission is still the same.

When I first sat and thought 'How can we turn this one-off Hail Mary for Mila into a mainstream way of helping millions?', I saw 3 major hurdles ahead: the science, the regulatory path and the viable, scalable business model. But with time, I realized that science was no longer the limiting factor - Tim and others proved that amazingly they could do this over and over again across different diseases and more recently different therapeutic modalities. But each time required an equally if not more herculean attempt. So it was the regulatory path that I focused on. I worked closely with Daniel O'Connor, Parker Moss and others to launch an infrastructure pilot in the UK - the Rare Therapies Launch Pad - to model what a new system could look like to meet this new paradigm. After years of work, both the UK and US regulators finally signaled at the end of last year an intention to move from Product to Process Approval - the critical shift we've been fighting for.

Now, I'm focusing on the last hurdle - the business and reimbursement model desperately needed to remove the weight from the shoulders of parents who feel hope for their children depends on whether they drive the work toward a treatment. Incredible parents and physicians have been working together to raise millions and become drug developers, one dying child at a time. But it's an unsustainable, unscalable model that's unethical for parents like me.

EOM and our team are determined to show that there is a different way to bring treatments to patients at scale - industrializing individualized medicines through Process Approval and reimbursement, and working closely with hospitals. Our groundbreaking treatment in London last week is just the beginning of what we believe will get us there and pave the way for many others.

The road that lies ahead offers big challenges, but even bigger opportunities.

Thank you again to The Economist and Natasha Loder for putting out this Podcast today that captures the next chapter in my mission from Mila to Millions.

PODCAST - https://www.economist.com/podcasts/2026/01/21/custom-cures-tailor-made-drugs-for-rare-diseases

01/19/2026

A BIG thank you to The Economist and Natasha Loder for shining a light on a very exciting moment for families like mine. Last week, a 15 year old girl with a fatal genetic disease received the UK's first individualized medicine giving her a second chance at life. She's the age Mila would have been today. In a way, I handed her family the hope I once had.

But this treatment also represents the beginning of a HUGE change - the lead-up to Process Approval and eventual reimbursement, where companies join the race and the flywheel starts spinning, connecting exponentially more children to potentially life saving treatments.

This therapy was developed by the incredible team at EveryONE Medicines - the biotech Tim Yu and I founded to prove individualized medicines can be made and delivered at scale - working closely with our close partner, Great Ormond Street Hospital and Charity, where this girl was treated. All of this happened in the context of the Rare Therapies Launch Pad, the pilot program many of us started to demonstrate the desperately needed system change. And the Medicines & Healthcare products Regulatory Agency (MHRA) played a critical role in giving the green light to an entirely new Master Protocol trial, crossing diseases, mutations and medicines...

"The (MHRA) has agreed that patient A and ten other children with fatal or life-threatening neurodegenerative conditions can be treated with custom drugs under a new "master protocol” that is intended to standardise trials for the treatment of groups of genetic conditions within a single framework. It thus tests a way of making drugs rather than assessing a single medicine. Lawrence Tallon, the MHRA ’s head, said it was the 'start of what is a very, very exciting future for the treatment of genetic diseases'.... The goal of the trial, says the MHRA, is to show that, with such a standard set of procedures, it can grant a 'process approval' for making these ASOs."

This is the change I've been fighting for...

ECONOMIST Article - https://www.economist.com/science-and-technology/2026/01/19/treatment-of-a-teenager-with-an-ultra-rare-condition-is-a-medical-milestone

01/16/2026

Great to hear that new U.S. Food and Drug Administration guidance on scalable access to individualized medicines - crossing modalities and Centers - will be coming out very soon in the US - as announced at the JPM panel this week by Vinay Prasad, MD MPH and Tracy Beth Høeg, MD, PhD.

In support of the FDA's recent 'Plausible Mechanism' paper in the , a number of us co-authored a paper in Molecular Therapy, 'Response to the FDA’s proposed pathway for individualized genetic therapies' to further imagine what a new standardized process-based approval pathway might look like. I'm hopeful that the key points we propose will be included in the final guidance.

PAPER - https://www.cell.com/molecular-therapy-family/molecular-therapy/abstract/S1525-0016(25)01055-X

01/08/2026

A big thanks to Becky Quick for launching CNBC Cures! And a special thank you to Scott Gottlieb for focusing on the incredible hope that individualized medicines offer the millions like Mila and Becky's daughter Kaylie. I appreciate the shout out!

Former FDA Commissioner Dr. Scott Gottlieb joins 'Squawk Box' to discuss the ongoing research and efforts at the FDA to find cures for rare diseases.