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Rockland teen organizes fundraiser to help others battle rare disease after own experience
A teen from Rockland County wants to help others battling rare diseases after his own fight. Carlos "Coca" Bernard has ...
One family, two battles: Sterling family faces stroke recovery, rare disease diagnosis
A Sterling family is seeking community support after a "perfectly normal day" changed their lives forever. Jeremy Hunter suffered a severe stroke May 20 that resulted in emergency surgery to remove a ...
Research Moves Slowly. Rare Diseases Don't—So Patients Aren't Waiting
Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?
Penn and CHOP open a center for a rare genetic condition, thanks to a $25 million donation
Michael Armellino first heard of Williams syndrome when his partner’s granddaughter, Maelyn, was born with the rare genetic condition 10 years ago. A simple internet search laid out that she could be ...
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How Patient Advocacy Is Guiding Rare Disease Drug Development
Bruce Leuchter, CEO, Neurvati, discusses the vital role of public participation in healthcare policy, highlighting how ...
Oconee County 6-year-old fights rare neurological disease
June is Alzheimer's and Brain Awareness Month. Colbie Schueneman, 6, is sharing her story with KIF1A, also known as KAND.
15 Medical Conditions So Surprising, You Won’t Believe They’re Real
When nerve cells don’t end up in the right place during brain development, it can cause periventricular heterotopia —a ...
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Cure Rare Disease Receives FDA Feedback on Limb-Girdle 2i/R9 Gene Therapy Program Following Successful Pre-IND Meeting
Cure Rare Disease (CRD), a nonprofit biotechnology organization developing genetic therapies for ultra-rare diseases, today announced the successful completion of a pre-Investigational New Drug ...
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Cure Rare Disease Receives FDA Feedback on Limb-Girdle 2i/R9 Gene Therapy Program Following Successful Pre-IND Meeting - BioSpace
WOODBRIDGE, Conn.-- (BUSINESS WIRE)--Cure Rare Disease (CRD), a nonprofit biotechnology organization developing genetic therapies for ultra-rare diseases, today announced the successful completion of ...
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Cure Rare Disease Receives FDA Feedback on Limb-Girdle 2i/R9 Gene Therapy Program Following Successful Pre-IND Meeting
Cure Rare Disease (CRD), a nonprofit biotechnology organization developing genetic therapies for ultra-rare diseases, today announced the successful completion of a pre-Investigational New Drug ...