The finalized guidance from the US FDA on drug development for rare diseases brings hope to individuals like Celine Dion battling stiff-person syndrome.
Seeking specialized treatment providers, utilizing resources like SurvivorNet Clinical Trial Finder, Clinicaltrials.gov, and PubMed, and exploring drug companies and “compassionate use” are crucial steps in dealing with rare diseases.
Academic centers and comprehensive care centers play a vital role, providing a team approach to care.
The guidance addresses challenges in rare disease development, emphasizing patient input and pediatric considerations.
Explore various options, including decentralized clinical investigations, natural history studies, and clinical trial eligibility.
For those diagnosed with rare diseases, there are resources available to navigate the journey and find hope in the evolving landscape of drug development.
