@stopbatten

Mila fought hard against Batten disease, a rare fatal condition with no cure. Her story is now giving new hope to millions with genetic disease.

 Boulder, CO
stopbatten.org/
Joined February 2017
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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.
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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.
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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 @BeckyQuick and @CNBC Cures for highlighting Wheeler story! CNBC Cures Article: cnbc.com/2026/09/10/rare-dis…

A rare disease is taking his vision. A gene therapy could help — if he can get it

A well-connected family’s fight to access a potential treatment for their son highlights the barriers most rare disease patients struggle to overcome.

cnbc.com
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A big thanks to @VWVLaw 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.
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I was truly honored to receive the PING Innovation Award - thank you Paul Gershlick and Jemma Gore-Booth! #PING2026 #mila2millions
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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.
LifeArc@lifearc1
Jun 24
We’re really pleased to welcome the Rare Therapies Launch Pad (RTLP) into LifeArc. RTLP has built a collaborative community focused on a shared challenge: how to turn advances in genetics into treatments that can reach individual patients.
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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.
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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.
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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.
MHRAgovuk
@MHRAgovuk
May 21
🔄 Today we launch our public consultation on a new framework to get life-changing treatments to rare disease patients faster. 🗣️ Julia Vitarello shares her daughter Mila's story. Watch, then have your say – closes 30th July 2026. 🔗 brnw.ch/21x2HaF
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The MHRA just announced the new Rare Disease Therapies Regulatory Framework which includes a section on individualised medicines recognising that the development of them may require a proportionate, risk-based approach to medicines regulation.
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This is a HUGE win and gives me more hope than ever for the children who follow Mila.
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The second stop on my European trip last week was London for the @lifearc1 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.
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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.
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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. #Mila2Millions
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