Kerala doctors successfully performed a life-saving liver transplant on a two-and-a-half-year-old child diagnosed with the ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read more at straitstimes.com. Read more at straitstimes.com.

Marci Shelman and Tristan Dederscheck’s “miracle” baby was diagnosed with neonatal Marfan syndrome. After four months in the ...

The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...

Dec 16 (Reuters) - U.S. Health ‌Secretary ​Robert F. Kennedy ‌Jr. on Tuesday added two rare ​genetic disorders, Duchenne ...

Rare genetic disorders affect more than 300 million people worldwide, with children making up 70% of those impacted, according to EURORDIS. These conditions often go undiagnosed for years due to ...

Wilson’s disease is an autosomal recessive disorder caused by ATP7B mutations, leading to copper accumulation in the liver, ...

Ultragenyx Pharmaceutical Inc. sank after the company’s experimental drug for a rare genetic bone disease failed to meet the ...

This year saw notable progress in head and neck cancers, Huntington's disease, personalized genetic therapy, and heart ...

BioMarin Pharmaceutical said on Friday it would acquire Amicus Therapeutics for about $4.8 billion, in the drugmaker's second ...