Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...
Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...
Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated ...
Please refer to the ASH 2025 online program for full session details and visit the Genetix booth (#1261) onsite.
13don 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.
The new intrathecal formulation means the one-shot gene therapy is now available to all SMA patients, regardless of their age ...
Biotechs in the charter city of Próspera aim to cure ageing, but experts question the feasibility of their goals.
Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only ...
Pharmaceutical Technology on MSN
Regeneron and Tessera join forces to forge in vivo AATD gene therapy
If approved, Regeneron and Tessera's TSRA-196 could become the first curative therapy to reach patients with AATD, who have ...
Novartis is pricing Itvisma at a wholesale acquisition cost of $2.59 million, a company spokesperson told Fierce Pharma. At ...
12don 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 ...
A study identified two key genes (CTNNA1 and BCL2L13) as suppressors in spread of colorectal cancer, revealing new strategies ...
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