Mila's Miracle

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.

[Mila's Miracle @stopbatten]

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

[Mila's Miracle @stopbatten]

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]

🔄 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.

🔗 https://t.co/p2fRgPuTjf

[Mila's Miracle @stopbatten]

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.

[Mila's Miracle @stopbatten]

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

[Mila's Miracle @stopbatten]

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.

[Mila's Miracle @stopbatten]

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.

[Mila's Miracle @stopbatten]

The system has been the limiting factor, but the growing willingness to accept a new mindset gives me hope.

#mila2millions

[Mila's Miracle @stopbatten]

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.

[Mila's Miracle @stopbatten]

I started with a presentation in Amsterdam at the @_knaw (Royal Netherlands Academy of Arts and Sciences) Symposium on Science & Sustainable Medicine where the message was clear - we're living in a unique time when we have incredible science to help patients.

[Mila's Miracle @stopbatten]

This Is Today: https://t.co/mKncsDkNdr

Featured Family: Sims Family
Photo Credit: Ulli Bonnekamp

#TeamCharlie #Mila2Millions #RareAsOne #ThisIsToday

[Mila's Miracle @stopbatten]

Bath time is a special time for Charlie and his dad Matt. A quiet moment together at the end of a long day. But Charlie can’t move independently anymore, so his dad has to carry and lift him often, including in and out of the bath.

[Mila's Miracle @stopbatten]

In his mind, there’s no choice but to push through it… because there are only so many more of these small moments of joy he might have left with his son.

[Mila's Miracle @stopbatten]

It's truly unique to sit in a relatively small room with many of the the country's leaders in the space, including in government, industry, and investment, who are so motivated to work together to build a better system for access to medicines... and in turn make a global impact.

[Mila's Miracle @stopbatten]

It was an honor to speak at the @MHRAgovuk's event at the @royalsociety in London this week alongside Lawrence Tallon and other leaders committed to making the UK the place for innovation and investment in health.