Spinal Muscular Atrophy
📧 Sign up to get email alerts when something new happens for Spinal Muscular Atrophy.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS trial_result · 2022-01-11Clinical Trial Update: Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies [Phase 3]
Phase 3 trial of a single-dose gene replacement therapy for spinal muscular atrophy type I with 1-2 SMN2 copies has completed and posted res…
-
CLINICAL_TRIALS trial_result · 2022-01-11Clinical Trial Update: Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 [Phase 3]
Phase 3 trial update evaluating intravenous Zolgensma (Onasemnogene abeparvovec-xioi) in presymptomatic spinal muscular atrophy patients wit…
-
CLINICAL_TRIALS trial_result · 2021-06-15Clinical Trial Update: A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants [Phase 2]
Phase 2 trial evaluating risdiplam in Type 2 and 3 Spinal Muscular Atrophy completed with results posted in 2021.
-
FDA approval · 2021-04-30FDA Approved: EVRYSDI — Supplemental Application (Genentech, Inc.)
The FDA approved a supplemental application for EVRYSDI (risdiplam) from Genentech, Inc.
-
CLINICAL_TRIALS trial_result · 2021-04-02Clinical Trial Update: Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 [Phase 3]
A Phase 3 trial of a single-dose gene replacement therapy for Spinal Muscular Atrophy Type 1 has completed, with results posted in 2021. The…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
EMA approval · 2021-02-26First oral treatment for spinal muscular atrophy (SMA) recommended for approval
The European Medicines Agency recommends approval of the first oral treatment for spinal muscular atrophy, marking a significant milestone i…
-
CLINICAL_TRIALS trial_result · 2021-02-21Clinical Trial Update: A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function [Phase 1]
Phase 1 pharmacokinetics and safety study evaluating hepatic impairment on risdiplam after a single oral dose, comparing impaired participan…
-
CLINICAL_TRIALS trial_result · 2021-01-08Clinical Trial Update: Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy [Phase 2]
A Phase 2 trial of risdiplam in infants with Spinal muscular atrophy completed, reporting safety, tolerability, PK/PD and efficacy with resu…
-
CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Flu Vaccine Study in Neuromuscular Patients 2011 [Phase 4]
A Phase 4 clinical trial update on a flu vaccine study in pediatric neuromuscular disease patients has completed, with results posted in 202…
-
CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants [Phase 1]
Phase 1 study evaluating drug-drug interaction between risdiplam (multiple dose) and midazolam in healthy participants with spinal muscular …
-
CLINICAL_TRIALS trial_result · 2020-08-31Clinical Trial Update: A Study of CK-2127107 in Patients With Spinal Muscular Atrophy [Phase 2]
A Phase 2 clinical trial of CK-2127107 in patients with spinal muscular atrophy has completed with results posted.
-
FDA approval · 2020-08-07FDA Approved: EVRYSDI — Original Application (Genentech, Inc.)
The FDA granted original approval for Evrysdi (risdiplam) from Genentech for the treatment of spinal muscular atrophy.
-
CLINICAL_TRIALS trial_result · 2020-07-16Clinical Trial Update: Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 [Phase 3]
Phase 3 trial results for a gene replacement therapy in spinal muscular atrophy type 1 were completed, with results posted in July 2020. The…
-
FDA approval · 2020-06-16FDA Approved: SPINRAZA — Supplemental Application (Biogen Inc.)
The FDA approved a supplemental application for Spinraza (nusinersen) filed by Biogen, indicating an expanded or updated labeling.
-
EMA approval · 2020-03-27New gene therapy to treat spinal muscular atrophy (corrected)
The EMA announces a new gene therapy for spinal muscular atrophy, marking a significant regulatory milestone for treatment of the disease.
-
CLINICAL_TRIALS trial_result · 2020-01-27Clinical Trial Update: A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). [Phase 2]
Phase 2 trial assessing safety and tolerability of nusinersen (ISIS 396443) in participants with spinal muscular atrophy was terminated; res…
-
CLINICAL_TRIALS trial_result · 2019-10-02Clinical Trial Update: A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients [Phase 1, Phase 2]
Pilot therapeutic trial evaluating hydroxyurea in Type II and Type III spinal muscular atrophy patients completed.
-
CLINICAL_TRIALS trial_result · 2019-10-02Clinical Trial Update: A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients [Phase 1, Phase 2]
Pilot therapeutic trial evaluating hydroxyurea in patients with Type I spinal muscular atrophy (Phase 1/Phase 2) completed with results post…
-
CLINICAL_TRIALS trial_result · 2019-08-09Clinical Trial Update: A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) [Phase 2]
Phase 2 study evaluating long-term safety, tolerability, and effectiveness of olesoxime in patients with spinal muscular atrophy has complet…
-
FDA approval · 2019-06-17FDA Approved: SPINRAZA — Supplemental Application (Biogen Inc.)
FDA approved Biogen's supplemental application for Spinraza (nusinersen). Spinraza is used to treat spinal muscular atrophy.
-
CLINICAL_TRIALS withdrawal · 2019-02-15Clinical Trial Update: Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting [NA]
Clinical trial studying feasibility to reliably measure functional changes in nonambulant neuromuscular patients has been withdrawn.
-
CLINICAL_TRIALS research · 2019-01-17Clinical Trial Update: Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy
Clinical trial update examining the reliability and validity of the ACTIVE-mini movement assessment for infants with spinal muscular atrophy…
-
CLINICAL_TRIALS trial_result · 2018-10-30Clinical Trial Update: A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) [Phase 2]
Phase 2 clinical trial evaluating nusinersen (ISIS 396443) for efficacy, safety, and pharmacokinetics in infants with spinal muscular atroph…
-
FDA approval · 2018-10-10FDA Approved: SPINRAZA — Supplemental Application (Biogen Inc.)
The FDA approved Biogen's supplemental application for Spinraza (nusinersen).
-
FDA approval · 2018-05-14FDA Approved: SPINRAZA — Supplemental Application (Biogen Inc.)
The FDA approved Biogen's supplemental application for Spinraza (nusinersen), expanding/ updating its approved use.