FDA approves Rocket gene therapy for rare immune disorder
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The Food and Drug Administration approved a gene therapy for severe leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disease that leaves children vulnerable to life-threatening infections.
Reed Jobs is keen for his $1bn venture capital fund Yosemite to make a difference.
The 34-year-old runs an oncology-focused venture capital fund that manages more than $1bn in assets, and is in the UK looking for investment opportunities.
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SMA is a rare, inherited condition that causes progressive muscle weakness, affecting movement, breathing and swallowing. People living with spinal muscular atrophy (SMA) in England are set to gain routine access to two potentially life-changing treatments, following our publication of final…
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