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Mechanism of Action
RNA interference; Survival of motor neuron 2 protein expression stimulants
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
Yes
-
Spinal muscular atrophy
New Molecular Entity
Yes
Highest Development Phases
Marketed
Spinal muscular atrophy
Most Recent Events
04 May 2021
The UK National Institute for Health and Care Excellence (NICE) extends clinical eligibility of nusinersen to include non-ambulant Type III spinal muscular atrophy patients under managed access agreement
17 Apr 2021
Adverse events data from a part A of phase II/III trial in spinal muscular atrophy presented at the American Academy of Neurology (AAN-2021)
12 Apr 2021
Biogen completes an expanded access programme for infantile-Spinal muscular atrophy (In adolescents, In children, In adults) in Colombia, New Zealand and Turkey, USA, Australia, Canada, Germany, Italy, Sweden and in the UK prior to April 2021 (NCT02865109)
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