A Student’s Perspective: Lessons from the 2025 RARE Drug Development Symposium

On behalf of Harvard Undergraduate Rare Diseases and HackRare, Allison Wu was thrilled to be invited to attend the Global Genes’ 2025 RARE Drug Development Symposium in Cambridge, MA.

“During my first week as a Harvard College junior, I was delighted to be given the opportunity to attend the Global Genes RARE Drug Development Symposium. Between my classes in Organelle Biology and Biochemistry & Molecular Medicine, I enjoyed venturing to the Harvard Medical School area to see experts, advocates, patients, and families come together to discuss the development of treatments for rare diseases. I learned that progress in rare diseases depends on cross-disciplinary translation from bench research to analyses of large datasets and community engagement.

The opening session, featuring Dr. Sonia Vallabh, PhD, Director of Prion Therapeutic Science at the Broad  Institute, was particularly inspiring to me. How a lawyer returns to school to earn a PhD in biological and biomedical sciences and becomes a world expert in developing preventive drugs for prion disease demonstrates the drive that an individual with a family history of a disease can have. Dr. Vallabh watched her mother die of an undiagnosed dementia at 52 years of age and then learned that she had inherited the causal mutation, making her at risk for developing the disease as well. Because no preventative treatments or cures were available, Dr. Vallabh and her husband both retrained as scientists. Dr. Vallabh’s story shows the tremendous need for more research in rare diseases. The Prion Therapeutic Science initiative that Dr. Vallabh leads is developing biomarkers to help detect the disease and new preventive drugs, and she has started a Prion Registry to help people affected by or at risk for prion disease to participate in research.

Throughout the rest of the conference, it became clear to me that discovering treatments for rare diseases is possible, but because each rare disease is uniquely rare, it’s imperative that the rare disease community comes together and learns from each other.”

Read the full article published by Global Genes here.