Written by Holly Alyssa MacCormick You can do a lot with $10 million. Yet, when Jeffrey Randall Allen won the largest prize in game show history on Amazon Prime Video’s Beast Games there was only one thing he wanted to spend his $10 million on—research that might help find a cure for his son’s rare … Continue reading Winner of Beast Games becomes champion for his son’s rare disease
Rare Diseases
CIRM $4 million awarded to support innovative Gene Therapy for Rare Heart Disorder
Image Source: American Heart Association Journals The California Institute for Regenerative Medicine (CIRM) recently awarded $4 million to support the development of a one-time gene therapy for desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM), a rare and life-threatening genetic disorder. This preclinical award to Deborah D. Ascheim, MD, and her team at Rejuvenate Bio are … Continue reading CIRM $4 million awarded to support innovative Gene Therapy for Rare Heart Disorder
Turning Hope into Reality: How a Genetic Therapy Helped Connor
Photo credit: n-Lorem Connor Dalby is a 15-year-old teenager who loves music and the beach, but his journey has been far from ordinary. Diagnosed with a rare mutation in the SCN2A gene, his life has been filled with challenges. From the time he was just a few weeks old, he had trouble sleeping and struggled … Continue reading Turning Hope into Reality: How a Genetic Therapy Helped Connor
Joining the movement to fight rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST It’s hard to think of something as being rare when it affects up to 30 million Americans and 300 million people worldwide. But the truth is there are more than 6,000 conditions – those affecting 200,000 people or fewer – that are considered rare. Today, … Continue reading Joining the movement to fight rare diseases
The Stem Cellar 