Mila's Miracle Foundation to Stop Batten

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Mila's Miracle Foundation to Stop Batten Mila fought hard against Batten disease. Now her story is giving hope to millions w. genetic disease.

Help us raise $4M to fund research and start a clinical trial to halt degeneration in children facing Mila's type of Batten, and work towards a cure.

One year into Mila receiving milasen at Boston Children's Hospital, I realized how hard it would be to get Mila to Bosto...
10/09/2026

One year into Mila receiving milasen at Boston Children's Hospital, I realized how hard it would be to get Mila to Boston every few months. Her team at B*H was incredible, but we needed to move her treatment close to home. I asked the doctors if they knew of anyone at Children's Hospital Colorado who they would trust with their own daughters lives - and they immediately said Dr. Scott Demarest.

Before I knew it, Scott and his colleagues had flown to Boston and were learning the ropes from the B*H team. Both hospitals worked closely to make the transfer with the FDA, and within months we were driving just one hour to get her dosed and home before dinner.

At our first appointment, Scott looked at Mila - not me - and asked her how she was doing. She couldn't see or speak then, but she turned to him and listened.

Since Mila, Children's Hospital Colorado has rapidly built a Precision Medicine program and Scott has grown with it to become their leader. Last week, I was so moved to be invited to honor him being named the Precision Medicine Chairholder, one of the highest honors in academic medicine.

I can't think of anyone more deserving. So excited to watch Scott help lead this new field!

10/09/2026

When Talia was 9 years old, her mom, Jocelyn, started fighting to save her daughter’s life. Within just 2 years, the scientists she had gathered had solid proof that their gene therapy might be able to stop her disease, but they lacked the millions of dollars needed to bring it to patients.

Tal used to ask when her 'muscle medicine' would be ready, but as the years passed and the numerous attempts to engage hospitals and companies failed, she stopped asking for it.

Today, Talia is 20 years old and her mom is still fighting. Talia has lost so many physical abilities but she is still thriving—she goes to college, hangs out with friends and family, and hikes in the woods in her wheelchair. Just recently, the treatment has been made and finally given the ok for patients. For Talia, this might mean getting better in certain ways - restoring some of her muscle function in her lungs, legs, arms or hands. Or it might mean just not getting worse. Either would be a huge win for Talia and her family.

As Jocelyn prepares for the big day she’s been fighting for, she picks up a photo of Talia at 12 years old - she's wearing a sparkly green dress standing over her science desk at school, her hands resting on the table as she looks down into a microscope. She's breathing on her own, standing on her own, using her arms and hands on her own. Jocelyn can't help but think about what Talia's life could have been if she had found the money and had her daughter treated then.

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

Photo/Video Credit: Ulli Bonnekamp & Kira Upin

Eleven years ago, Talia was diagnosed with a deadly, progressive genetic disease. Since then, her parents have been by h...
10/02/2026

Eleven years ago, Talia was diagnosed with a deadly, progressive genetic disease. Since then, her parents have been by her side every single day. Her father, John, is the quiet steady force behind everything. Her mother, Jocelyn, is the one communicating with researchers, asking whether Talia's legs are in a comfortable position, checking her ventilator at night. John stops to ask Talia if she had a good day, thinking ahead to what game they could play next. Jocelyn lays awake at night worrying about what tomorrow may bring.

Together, they’ve been working tirelessly to create a beautiful life... one day at a time.

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

Featured Family:

Photo Credit: Bonnekamp

When Talia was 9, she was diagnosed with CMT4J, a progressive genetic disease similar to ALS that affects just 1 in 1 mi...
09/30/2026

When Talia was 9, she was diagnosed with CMT4J, a progressive genetic disease similar to ALS that affects just 1 in 1 million people. As a little girl, Talia walked through the halls of her school with a walker or a helping hand. She was able to feed herself and was famous for her huge hugs. Now her nerves are dying, cutting off connections to her muscles, including her lungs. She needs a wheelchair to get around. She has lost the use of her arms and hands. She needs a ventilator to breathe.

But Talia’s voice has become her superpower. It helps her to navigate life, advocate for herself, and break out into 80’s songs in her rock band to show the world who she is.

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

Featured Family:

Photo Credit: Bonnekamp

Soon after Mila began receiving milasen, I met Judy Stecker whose son Wheeler had been diagnosed at birth with a form of...
09/16/2026

Soon after Mila began receiving milasen, I met Judy Stecker whose son Wheeler had been diagnosed at birth with a form of Batten disease. She was working for the US government and knew the right people at the FDA, HHS and the White House. There were numerous treatments in the pipeline. And she had a healthy baby years away from his first symptoms. I was sure that Wheeler would be the one whose disease would be stopped in time.

But today Wheeler is 7 years old and he's no closer to a treatment than he was at birth. He's rapidly loosing his vision by the month and he's stopped riding his scooter. He's scared and confused about what's happening to him.

There is a promising gene therapy on the horizon... and I have great hope that Wheeler can finally gets access and it comes in time.

Thank you Becky Quick and CNBC Cures for highlighting Wheeler story!

CNBC Cures Article: https://www.cnbc.com/2026/09/10/rare-disease-is-taking-his-sight-access-to-a-treatment-is-hard-to-get.html

06/25/2026

A big thanks to VWV Law for inviting me to present remotely at the PING 2026 Meeting in London to more than 150 senior leaders who came together to tackle the question of what it will take for the UK to lead Europe in life sciences by 2030. I was truly honored to receive the PING Innovation Award - thank you Paul Gershlick and Jemma Gore-Booth!

It's an exciting day! The Rare Therapies Launch Pad is moving into LifeArc, a powerhouse organization with an aligned mi...
06/24/2026

It's an exciting day! The Rare Therapies Launch Pad is moving into LifeArc, a powerhouse organization with an aligned mission to change the system to meet the reality of rare diseases.

The RTLP was created to move beyond discussions and actually demonstrate the system that needs to be in place for patients to access individualized medicines at scale. This next phase of the RTLP is critical as the UK regulatory framework opens up, and no one is better suited than LifeArc to ensure that key organizations work together with urgency to show the world how this can be done.

05/22/2026

Three years ago, I decided to bet on the UK as the place that would demonstrate a new system for accessing individualized medicines at scale.

I read that former prime minister David Cameron lived a similarly tragic story to mine with his son Ivan and assumed it must have changed the country in some way, and it did. I learned about Genomics England which David helped start and their country-wide Whole Genome Sequencing which most recently focused on newborns. I was introduced to a healthcare system that housed clinical and genomic data for nearly all patients in the UK. And I teamed up with Daniel O'Connor, Parker Moss and others to start the Rare Therapies Launch Pad to help push the system change we desperately need.

But the real change had to start with the Medicines and Healthcare products Regulatory Agency. They've had the right mindset, but in the last year I've watched Lawrence Tallon, Julian Beach, Dr Charlotte McIntyre MD, PhD and their team really step it up and actively work towards a new regulatory pathway that enables faster, reimbursable access to today's science for the millions who fall into small groups by disease or by medicine.

Today, I smile at my decision to place my bet on the UK. The MHRA just announced the new Rare Disease Therapies Regulatory Framework which includes a section on individualized medicines in which "a medicinal product would be licensed on the basis that pre‑defined elements of the active substance may be customised for a patient based on their unique characteristics, provided the developer demonstrates quality, safety, and efficacy with a focus on the process by which such individualised variants are designed and produced. Developers of individualised medicines would therefore not be expected to apply for a separate marketing authorisation for each individualised and unique batch of product. The MHRA recognises that the development of such individualised medicines may require a proportionate, risk-based approach to medicines regulation."

This is a HUGE win and gives me more hope than ever for the children who follow Mila.

The second stop on my European trip last week was London for the LifeArc Translational Science Summit. It was truly an h...
05/21/2026

The second stop on my European trip last week was London for the LifeArc Translational Science Summit. It was truly an honor to give the closing keynote, after a great day of talks kicked off by Reed Jobs and Dr Sam Barrell CBE.

LifeArc is playing a critical role in bringing together and supporting organizations in the rare disease space to forge new paths that improve and save lives. The UK is leading the way so LifeArc is in a perfect position to help push the system so children like Mila can access treatments they urgently need.

I've just returned from a long, but very positive week in Europe, where individualized medicines took center stage in di...
05/20/2026

I've just returned from a long, but very positive week in Europe, where individualized medicines took center stage in discussions about helping millions of patients access today's incredible science.

I started with a presentation in Amsterdam at the KNAW - Koninklijke Nederlandse Akademie van Wetenschappen (Royal Netherlands Academy of Arts and Sciences) Symposium on Science and Sustainable Medicine where the message was clear - we're living in a unique time when we have incredible science to help patients. The system has been the limiting factor, but the growing willingness to accept a new mindset gives me hope.

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