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.