It’s nearly Rare Disease Day and we’re getting ready to demonstrate our support for the millions of people living with rare diseases. Will you #ShowYourStripes and help raise awareness of the impact rare disease has on people’s lives? #RareDiseaseDay

Today is #RareDiseaseDay, and IGT is helping to advocate for a policy and regulatory framework that will help #raredisease patients access life changing #genetherapies. Learn more at gene-therapies.org

I am very excited to share Institute for Gene Therapies Value Principles. These were developed in collaboration with our Advisory Councils to ensure that the profound impact on many aspects of patients’ and their families’ lives are taken into account when determining value.

A really cool story about the incredible impact and promise of #genetherapy for patients and their families.

IGT sent a letter to Congress applauding Rep. Brett Guthrie Rep. Anna G. Eshoo Rep. Mariannette Miller-Meeks, M.D. Rep. Jake Auchincloss 🟧 & Rep. Scott Peters for their sponsorship of the Medicaid VBPs for Patients Act, or the MVP Act. (1/2) gene-therapies.org/post/igt-congr…

Thank you Lauren Holder for having me on the Help4HD Podcast! Enjoyed the discussion about what #genetherapy could mean for Huntington’s disease patients. Thank you for your leadership and advocacy!

Today Energy and Commerce Committee / Energy & Commerce Democrats will markup the #MVPAct – big thanks to my former colleagues Rep. Brett Guthrie Rep. Anna G. Eshoo Rep. Mariannette Miller-Meeks, M.D. Rep. Jake Auchincloss 🟧 John Joyce, M.D. Rep. Scott Peters for sponsoring this bill. Learn more about the MVP Act here: gene-therapies.org/post/what-they…

A great conversation about one of many patients’ journeys and the importance of ensuring access to #genetherapy. Take a listen to an outstanding Hill brief and discussion.

Many serious & life-threatening disease populations are very rare, affecting as few as one patient globally. IGT advocates for modernized, flexible policy & regulatory frameworks to help advance #genetherapies for #patients living with #unmetmedicalneed. bit.ly/45Y6OnF

I w/ emphasized Ambassador Klymenko how the US stands w/ Ukraine

Exciting news for patients with todays announcement

Join us in person or tune in virtually – I’m excited to dive deep into the policy and regulatory world of #genetherapy with this all-star panel.

A huge thanks to American Enterprise Institute & Brian Miller, M.D., M.B.A., M.P.H. for hosting a dynamic discussion on #genetherapy this week. Grateful to be a part of this incredible panel. If you missed our discussion, you can watch the full recording here: gene-therapies.org/post/aei-panel…

May is #HuntingtonsDiseaseAwarenessMonth and in support of our friends in the Huntington's community we wanted to highlight this important perspective from our wonderful PAAC member Lauren Holder: thehill.com/opinion/congre…

The mission of International Republican Institute and House Democracy Partnership is more important now than ever. I enjoyed being with my colleague Nick Rahall in Timore Leste

#newbornscreening is so important for patients - especially those excited about the promise of #genetherapy

Valentine's Day is also IGT's 5th anniversary, so I wrote a quick post reflecting on IGT's progress over the past five years and our continued commitment advocating for patient access to gene therapies.

I was honored to join CoalitionHemoB podcast to discuss how #genetherapy is transforming treatment for #rarediseases - and how we can help advocate for policies that ensure patient access. Listen here: hemob.org/podcast/episod…

“We are starting to see glimmers of hope where we didn’t see anything before but it is now time to kick the door open.” Children's Hospital’s Dr. Kiran Musunuru. #FacesofGeneTherapy

A big thank you to everyone who came to Capitol Hill yesterday for our Congressional Briefing on Promoting Innovation and Access to Cell and Gene Therapies, co-hosted with Institute for Gene Therapies. 1/2