Ladders to Cures Accelerator Symposium: Session 2 | Actionable Mechanisms Driving Rare Disease

Session 2: Actionable Mechanisms Driving Rare Disease
Chair: Jillian Shaw

Somatic Repeat Expansion as a Shared Therapeutic Target for Repeat Expansion Disorders
Ricardo Mouro Pinto

Characterization of the functional and clinical impacts of CACNA1A de novo missense variants
Jen Pan

Ó³»­´«Ã½ening the Role of Survival of Motor Neuron Protein
Lee Rubin

We are excited to invite you to the second annual Ladder to Cures Symposium | Reaching New Heights whose goal is to share progress in scalable technologies and advances in biology leading to treatments and cures for patients with rare genetic diseases. This hybrid all-day event will be held on May 16th, 2025 in the Ó³»­´«Ã½ Auditorium and streamed online. Anyone with an interest in rare disease research is invited to attend.

Building on Ó³»­´«Ã½â€™s strengths of scaling scientific efforts to accelerate progress, this symposium will convene scientists, physicians, patients/patient-led foundations and advisors from the biopharma and regulatory space to join the conversation.

For more information visit: /L2C

Ladders to Cures Accelerator Symposium: Reaching New Heights