Advances in stem cell biology and immune cell engineering bring diabetes cell therapies closer to reality.

Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple effect is already being felt across biopharma.

Childs Cure co-founders Jainu and Shruti Jogani founded the organization to address the genetic causes of rare diseases, ...

The nonprofit’s stem cell therapy was able to completely restore the cornea in one of the 14 patients on the Phase I/II trial ...

Hearing impairment affects millions globally, with genetics playing a major role in infant hearing loss. Early screening and ...

A bill banning gene-based vaccines, like some forms of the COVID-19 vaccine, received approval from a Senate subcommittee ...

Dr. Guru Sonpavde emphasized the importance of better understanding how genetic mutations influence the treatment of cancer ...

BLOOM (TAMPA) -Hormone Replacement Therapy (HRT) can be a game-changer for those struggling with hormone imbalances, but ...

Bone density dramatically declines as we age, and is accelerated among those who don't perform resistance exercise or ...

Interim data from Dose 1 PBFT02 demonstrated durable, elevated CSF PGRN and early evidence of reduction in plasma NfL levels, a disease ...

Sarepta Therapeutics hits 52-week lows despite strong earnings and growth. Read why investors should view SRPT as a high risk ...

The changes at Spark follow Roche's decision last year to halt development of a gene therapy for hemophilia A that was the ...