Patients Rising, Cystinosis Awareness
Written by K.M. I have never given much thought to how I would die. Perhaps because my health and longevity has been laid out for me since infancy. Maybe I…
Written by K.M. I have never given much thought to how I would die. Perhaps because my health and longevity has been laid out for me since infancy. Maybe I…
In the last decade, NAFLD in pregnancy has nearly tripled in concert with the worldwide increase in diabetes. Mayo Clinic reports that NAFLD in general affects one-fourth of the…
Marsha Calloway-Campbell lives a full life as a lawyer, business owner, wife to her husband Armaray, mother to her three adult daughters, and “Naun” to her toddler grandson. But her…
Gene therapy has seemed out of reach for decades. Yet advocates have considered it a way to have damaged or missing genes restore dystrophin and other proteins. Doctors and advocates…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
For many rare diseases, gene therapy, not traditional therapeutics, is perhaps the only means to significant relief and cure. However, the path to gene therapy has been strewn with obstacles…