NICE recommends Sprinraza for 5q spinal muscular atrophy
The recommendation is for infants, children and adults with 5q spinal muscular atrophy and will be available through a scheme known as a Managed Access Agreement.
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Tag: nusinersen
Five therapies endorsed for use on NHS Scotland
A new treatment option for the very rare condition infantile onset 5q spinal muscular atrophy (type I SMA) is among five medicines that have been endorsed by Scottish cost regulators for use on the NHS.
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EU clears Biogen’s Spinraza for rare spinal disorder
EU regulators have issued clearance for the first therapy to treat spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement.
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US licenses first drug for rare spinal disorder
US regulators recently approved the first therapy to treat spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement.
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- Lindus Health and Quotient Sciences announce strategic partnership to accelerate drug development
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