Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2024-10-09Clinical Trial Update: Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
This completed clinical trial sponsored by Novartis analyzes therapeutic management and the use of resources and costs associated with spina…
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CLINICAL_TRIALS trial_result · 2024-09-27Clinical Trial Update: Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular Disease in Their Natural Environment [NA]
A completed clinical trial evaluating the safety and usability of a robotic gait device for children and adolescents with neurological or ne…
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CLINICAL_TRIALS research · 2024-09-24Clinical Trial Update: Ultrasonographic Muscle Assessment and Functional Scales in Spinal Muscular Atrophy
An active-not-recruiting clinical trial from Istanbul University Cerrahpasa is investigating ultrasonographic muscle assessment and function…
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1 [Phase 1, Phase 2]
This clinical trial update describes an ongoing Phase 1/2 study evaluating the safety and efficacy of EXG001-307 in patients with spinal mus…
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FDA label_change · 2024-09-17FDA Approved: EVRYSDI — Supplemental Application (Genentech, Inc.)
FDA approved a supplemental application for Evrysdi (risdiplam) by Genentech, updating the product label.
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CLINICAL_TRIALS trial_result · 2024-09-03Clinical Trial Update: Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients [Phase 2, Phase 3]
Phase 2 and Phase 3 clinical trials evaluated short-term and long-term treatment with 4-aminopyridine (4-AP) in ambulatory patients with spi…
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CLINICAL_TRIALS trial_result · 2024-08-28Clinical Trial Update: Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I [EARLY_Phase 1]
A completed early-phase 1 clinical trial evaluated the safety and efficacy of EXG001-307 in patients with Spinal Muscular Atrophy Type I.
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CLINICAL_TRIALS research · 2024-08-20Clinical Trial Update: Hippotherapy in Children With Spinal Muscular Atrophy [NA]
A clinical trial in the Czech Republic is evaluating hippotherapy as an intervention for children with spinal muscular atrophy; the study is…
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CLINICAL_TRIALS research · 2024-08-16Clinical Trial Update: Exploring Bulbar Function, Speech And Communication Development in SMA Type 1
A clinical trial is ongoing to study bulbar function, speech, and communication development in infants with spinal muscular atrophy type 1, …
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CLINICAL_TRIALS research · 2024-08-06Clinical Trial Update: Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients [NA]
This is an observational study update examining outcome measures and biomarkers in spinal muscular atrophy type III/IV patients. It is spons…
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CLINICAL_TRIALS research · 2024-07-23Clinical Trial Update: UK SMA Patient Registry
The UK Spinal Muscular Atrophy (SMA) patient registry sponsored by Newcastle University is currently recruiting.
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CLINICAL_TRIALS trial_result · 2024-05-31Clinical Trial Update: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) [Phase 4]
Phase 4 OFELIA trial evaluating intravenous AVXS-101 (onasemnogene abeparvovec) in pediatric spinal muscular atrophy has completed with resu…
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CLINICAL_TRIALS trial_result · 2024-04-29Clinical Trial Update: A BHI to Increase Hope Level and Stress Level of Parents With a CMC [NA]
This ClinicalTrials.gov update reports the completion of a study by The Hong Kong Polytechnic University evaluating a behavioral health inte…
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FDA approval · 2024-04-10FDA Approved: SPINRAZA — Supplemental Application (Biogen Inc.)
The FDA approved Biogen's supplemental application for Spinraza (nusinersen), indicating an expansion/modification to the product's labeling…
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CLINICAL_TRIALS trial_result · 2024-03-05Clinical Trial Update: A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy [Phase 2]
This clinical trial update reports results from a Phase 2 study of risdiplam in infants who are genetically diagnosed and presymptomatic for…
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FDA approval · 2024-02-29FDA Approved: EVRYSDI — Supplemental Application (Genentech, Inc.)
The FDA approved a supplemental application for EVRYSDI (risdiplam) from Genentech, Inc.
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CLINICAL_TRIALS trial_result · 2024-02-07Clinical Trial Update: Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen [Phase 4]
Phase 4 trial update evaluating risdiplam in patients with spinal muscular atrophy previously treated with nusinersen.
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CLINICAL_TRIALS trial_result · 2024-02-01Clinical Trial Update: WREX Outcome Study
Clinical trial update for the WREX Outcome Study (NCT02218593) completed with posted results.
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CLINICAL_TRIALS trial_result · 2024-01-05Clinical Trial Update: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) [Phase 3]
Phase 3 trial results for AVXS-101 (OAV101) intravenous gene therapy in pediatric spinal muscular atrophy, with results posted.
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FDA approval · 2023-10-03FDA Approved: EVRYSDI — Supplemental Application (Genentech, Inc.)
FDA approved a supplemental application for EVRYSDI (risdiplam) from Genentech, Inc.
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FDA approval · 2023-02-17FDA Approved: SPINRAZA — Supplemental Application (Biogen Inc.)
FDA approved a supplemental application for Spinraza (nusinersen) submitted by Biogen. This action updates the drug's labeling and may expan…
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CLINICAL_TRIALS trial_result · 2023-02-16Clinical Trial Update: Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy [Phase 1]
Phase 1 clinical trial evaluating intrathecal administration of onasemnogene abeparvovec-xioi for spinal muscular atrophy was terminated, wi…
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CLINICAL_TRIALS trial_result · 2022-11-17Clinical Trial Update: An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy [Phase 2]
This document reports the completion of a Phase 2 active-treatment trial of SRK-015 in spinal muscular atrophy (types 2 and 3) with results …
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FDA label_change · 2022-09-21FDA Approved: EVRYSDI — Supplemental Application (Genentech, Inc.)
FDA approved a supplemental application for Evrysdi (risdiplam) from Genentech, leading to changes to the product label.
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FDA approval · 2022-05-27FDA Approved: EVRYSDI — Supplemental Application (Genentech, Inc.)
The FDA approved a supplemental application for Evrysdi (risdiplam) from Genentech, expanding the approved labeling for the drug. This actio…