Synonyms 2型脊髓性肌萎缩, Intermediate spinal muscular atrophy, Late infantile spinal muscular atrophy + [23] |
Introduction An autosomal recessive condition caused by mutation(s) in the SMN1 gene, encoding survival motor neuron protein. It is characterized by onset between 3 and 15 months of age, and is intermediate in terms of severity between spinal muscular atrophy (SMA) type I and SMA type III. |
Target |
Mechanism SMN1 gene stimulants |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhaseApproved |
First Approval Ctry. / Loc. US |
First Approval Date24 May 2019 |
Target |
Mechanism MSTN inhibitors |
Active Org. |
Originator Org. |
Inactive Indication- |
Drug Highest PhasePhase 3 |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Target |
Mechanism SMN1 gene stimulants |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePhase 1/2 |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Start Date31 May 2024 |
Sponsor / Collaborator |
Start Date15 Dec 2023 |
Sponsor / Collaborator |
Start Date01 Aug 2023 |
Sponsor / Collaborator |