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Addressing unmet needs for inherited neuromuscular diseases -
Press Release
Novartis announces partnership with the American Society of Hematology to fight sickle cell disease in Sub-Saharan Africa
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Breaking down barriers for patients with sickle cell disease -
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Discovery gives cystic fibrosis researchers new direction -
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When tiny brains are best for brain research -
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Fixing what’s lost in translation -
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Eye disease tracked to a glitch in starburst cells -
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Electrical brainstorms traced to genetic mutations -
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Small viruses could accelerate cell and gene therapy research -
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Rewriting the playbook for clinical testing in a rare disease -
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Rare speed on a rare disease -
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Novartis researches SMA’s genetic roots