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
Joined February 2017
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Four years ago, I came to the UK with a belief that we could demonstrate a new, scalable way for patients like Mila to access the incredible science available today through individualised genetic medicines.
Last month, I spoke with Grace Hampson & Amanda Cole from the @OHENews on their podcast 'A Dose of Economics' on the critical topics beyond regulation, on what HTA bodies and payers also need to change to bring individualised medicines to more patients.
Next month marks 10 years since Mila was diagnosed with Batten disease. It all started in Denver, Colorado, the same place where the @N1Collaborative just hosted their annual meeting.
Chloe, Dana, Arran, Tiziana, and Tomas represent who we are fixing the system for - to help patients, the next generation of new doctors and scientists must find a changed system in place that ensures that every child in need can access today's incredible science.
So the next Mila's can be found and treated in time so that they and their families never know life with disease.
#mila2millions
Talia's older sister, Teaghan, grew up watching her sister be diagnosed with CMT4J at just 8 years old. For Teaghan, there is no before or after. She only knows life as it is now: a slow path of loss and the ways that their family finds meaning within that.
At 23, Teaghan is a scientist, now living on her own. But she still has to balance the double-act of being a sister as well as a guardian; a friend as well as a caretaker.
Through all of this, Teaghan has acquired grit, and the understanding that grief and joy often walk hand in hand.
This Is Today: this-is-today.com/duff-famil…
Featured Family: Duff Family
Photo Credit: Ulli Bonnekamp
#Mila2Millions #RareAsOne #ThisIsToday #CureCMT4J
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 BCH was incredible, but we needed to move her treatment close to home.
Since Mila, @ChildrensColo 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.
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: this-is-today.com/duff-famil…
Featured Family: Duff Family
Photo Credit: Ulli Bonnekamp
#Mila2Millions #RareAsOne #ThisIsToday #CureCMT4J