Novel Approaches to Gene Therapies for Patients With Rare Genetic Diseases
Novel Approaches to Gene Therapies for Patients With Rare Genetic Diseases https://pediatricsnationwide.org/wp-content/themes/corpus/images/empty/thumbnail.jpg 150 150 Jessica Nye, PhD https://secure.gravatar.com/avatar/?s=96&d=mm&r=gAccording to the National Institutes of Health (NIH), nearly 7,400 diseases have been identified and classified as “rare” or “orphan” — “rare” because each afflicts fewer than 200,000 people in the United States and “orphan” because drug companies, unlikely to recoup research and development costs from such small patient populations, have historically found them too…