6don MSN
California boy, 3, receives first-ever gene therapy for his rare disease: ‘It’s just so exciting’
A California toddler is the first person in the world to receive gene therapy to treat his devastating disease.
Harlow is defying the odds after receiving groundbreaking gene therapy for a rare brain disorder. Her progress has been called "a miracle" by her family ...
5don MSN
3-Year-Old Boy with Rare Condition Amazes Doctors by Becoming World's First Gene Therapy Patient
Oliver Chu was born with a rare genetic condition called Hunter syndrome that impacts physical and mental development. The ...
The first child in history has successfully been treated with a new genetic therapy for an ultra-rare developmental defect ...
Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...
Novartis is pricing Itvisma at a wholesale acquisition cost of $2.59 million, a company spokesperson told Fierce Pharma. At ...
The FDA has approved Itvisma for the treatment of children two years and older, teens and adults living with spinal muscular ...
A new, one-time treatment could be the answer to cure Huntington's disease, a neurodegenerative disease long-considered ...
The 2025 Research Innovation Grants are supporting projects focused on biomarkers, risk factors, and treatments for ALS.
The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access ...
Treatment with an intramuscular hepatocyte growth factor reduced healing time and promoted full ulcer healing compared with placebo in patients with peripheral artery disease.
Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only ...
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