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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
12 Apr 2021
Biogen completes an expanded access programme for infantile-Spinal muscular atrophy (In adolescents, In children, In the elderly, In adults) in Colombia, New Zealand and Turkey , prior to April 2021 (NCT02865109)
03 Feb 2021
Biogen plans the phase III ONWARD trial for Spinal muscular atrophy (Intrathecal) in April 2021 (NCT04729907) (EudraCT2020-004708-32)
25 Apr 2020
Interim adverse events and efficacy data from a phase II Nurture trial in Spinal Muscular Atrophy presented at the 72nd Annual Meeting of the American Academy of Neurology (ANN-2020)
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