Tag: Rare genetic disorders

For the first time, scientists said they reached into the genome of a really sick kid and changed the unique misspelling in his DNA. It was a big deal, like seriously huge, and it...

Decades of NIH Research Reveals Insights on Rare Bone, Skin, and Endocrine DisorderPatients with fibrous dysplasia/McCune-Albright Syndrome often face challenges such as easily breaking bones, early puberty, and potential vision and hearing loss. For...