Buffalo Initiative

Today is #RareDiseaseDay, and I’m headed home after spending the week at the #BioNeuroscience meeting in Amsterdam. This is an annual summit of CEOs, global pioneers, investors and decision-makers in CNS neuroscience drug development. Kudos to the organizers (Christian Suojanen Amsterdam UMC - Amsterdam Neuroscience Broadreach Global) for ensuring patient-driven innovation for rare neurodevelopmental disorders was on the agenda. I’m always grateful for the opportunity to speak about the pivotal role patient groups play in accelerating research and improving care for our communities in forums like this one. But being able to share the story of the CACNA1A Foundation and the Buffalo Initiative alongside women who have inspired, guided and supported me on this journey since the very beginning was particularly meaningful for me (thank you Yael Weiss Allyson Berent Charlene Son Rigby). Getting the chance to spend time with Hans Schikan Juan Carlos Lopez, Vikram Sudarsan (Engrail Therapeutics) and Robert Thorne (Denali Therapeutics) was icing on the cake. While I missed the rare advocacy activities in DC this week, I know telling our stories of determination, urgency and progress in the face of daunting odds had an impact here. Onwards!

Available now at: https://lnkd.in/gr6em5Mr Thanks to Sunitha Malepati, CACNA1A Foundation, The Buffalo Initiative, OMSLIFE FOUNDATION, Principled Research Resources, L3C, and of course our host, Richard Juknavorian of Meeting You Where You're At Join us for a compelling episode of Rare Awareness Radio as we welcome Sunita Malipadi — attorney, advocate, and mother — who shares her powerful journey through the rare disease world. ?? Sunita's daughter was born with a mutation on the CACNA1A gene, leading to developmental challenges and a two-and-a-half-year diagnostic odyssey. Instead of giving up, Sunita turned her family’s experience into action. She joined the CACNA1A Foundation as vice president and founded the Buffalo Initiative, leading the charge in patient-driven rare disease research. For more information, please visit www.cacna1a.org/ In this episode, Sunita talks about: ?? The emotional rollercoaster of their diagnostic journey. ?? Why patient-led advocacy is essential for driving innovation. ?? The mission of the Buffalo Initiative to tackle the 'valley of death' in genetic medicine. ?? Her hopes for a more equitable and accessible research ecosystem. ?? Follow us for more inspiring stories from the world of rare disease advocacy. #RareAwarenessRadio #RareDisease #GeneticMedicine #CACNA1A #PatientAdvocacy #BuffaloInitiative #HealthcareInnovation

This is why the Buffalo Initiative was created — to support these efforts at scale.

I am deeply grateful to Kamal Menghrajani for the once-in-a-lifetime opportunity to be the opening "priority setting" speaker for the The White House Cell and Gene Therapy Forum, hosted by the White House Office of Science and Technology Policy yesterday. It was humbling to be in a room with 80 extraordinary individuals—executives from the most innovative biopharma companies, funders of groundbreaking work, federal leaders, and experts whose contributions I have long admired. As a mom and lawyer without formal scientific or technical training, I often question if I belong in rooms like this. To not only be invited but to have a voice in such critical discussions was profoundly affirming. Over the past five years of immersing myself in the rare disease drug development journey, I’ve realized something that’s really hard to believe: the biggest challenges in the field are no longer scientific—they are, as John F. Crowley put it, “man-made problems.” Patient communities are one of the most disruptive forces we have to overcome these challenges. Our voices, our stories, and the paradigms we’ve developed out of sheer necessity push leaders in the space to reflect on their role in a system that isn’t working for the 95% of rare diseases that lack an FDA-approved treatment and to do something about it. The solutions are within reach. Initiatives like the Buffalo Initiative are examples of how we can create new paradigms and realign existing models to meet the needs of patient communities. But this requires bold leadership, collective action, and a willingness to disrupt the status quo for the sake of the patients whose lives depend on it. We must embody a buffalo in a storm— face these challenges together as one herd and charge forward to a future where all communities have access to the treatments they so desperately need. ?? ?? #WhiteHouseCGT

Thank you Richard Juknavorian for the opportunity to share my story and the work we are doing at the Buffalo Initiative, a new collective impact paradigm focused on enabling patient communities to rapidly translate science into medicines.

Thank you Daniel Levine for the opportunity to speak with you about our work at the CACNA1A Foundation and what led me to launch the Buffalo Initiative, a collaborative philanthropy effort to scale community-led therapeutic discovery for childhood brain diseases. Listen to our conversation here: https://lnkd.in/eezHHNWN