In rare disease, urgency is often discussed in abstract terms — pipeline acceleration, regulatory timelines, translational gaps. But for some, urgency has a date attached to it. Yentli E. Soto Albrecht, PhD, is an MD-PhD student at the University of Pennsylvania whose life and work converged in August 2024 when her father — a 42-year educator in Lancaster, Pennsylvania — died from rapidly progressive ALS caused by the C9orf72 genetic mutation. He died fourteen months from hi

Rare disease communities understand fragility — not only biological fragility, but system fragility. For families navigating rare and medically complex conditions, crisis is rarely theoretical. It can be clinical, behavioral, logistical, and financial. It can surface at 2 a.m. in an emergency room unequipped for complexity. It can appear when a caregiver reaches burnout. It can emerge when fragmented systems fail to communicate across silos. While crisis infrastructure is oft

Heather F., co-founder of Level 5 Insights, is redefining healthcare market research with a mission rooted in empathy, inclusion, & advocacy

Explore how Rare360 empowers the rare disease community through advocacy and trust-building.

From personal experience to professional impact, Brittany Jež is transforming rare disease diagnostics for the hEDS community and beyond

A discussion with Mental Health for Rare Thought Leader JC Muyl.

Discussing the impact of clinical studies on patients’ lives and Michael’s perspective as a clinical trials operations specialist.