Trial reportNeurology2024
Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.
Erratum issued Registry-linked trialOpen access · hybridAbstract readRandomized Controlled TrialClinical Trial, Phase IIIClinical Trial, Phase II
In one paragraph
Trial report in Neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT03921528 (Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy), which is not on this map. Cited by 31 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
31citing papers in PubMed, 1 pooled it
15.8field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)
Who cites it
31 citing papers in PubMed, 1 synthesis or guideline pooled it, 36 citations in OpenAlex.
- Myostatin Modulation in Spinal Muscular Atrophy: A Systematic Review of Preclinical and Clinical Evidence.International journal of molecular sciences · 2025Pooled it
- Apitegromab for lean mass preservation during tirzepatide-induced weight loss: a randomized, double-blind, placebo-controlled phase 2 trial.Nature medicine · 2026Trial
- Antibodies to watch in 2026.mAbs · 2026Review
- Review
- Sarcopenia and Skeletal Muscle Dysfunction in Liver Cirrhosis: Clinical Perspectives of Myostatin Inhibition.Biomedicines · 2026Review
- Ten years of disease-modifying therapy in spinal muscular atrophy: lessons learned and future directions.Nature reviews. Neurology · 2026Review
- Development of pharmacological interventions for the treatment of sarcopenia.Annals of translational medicine · 2026Review
- Myostatin Research: From Molecular Understanding to Clinical Translation for Musculoskeletal and Metabolic Disorders.International journal of molecular sciences · 2026Review
- ActIIR inhibition improves motor outcome and preserves muscle fibers after experimental autoimmune neuritis.Acta neuropathologica communications · 2026Article
- Humans with function-disrupting variants in the myostatin gene (MSTN) have increased skeletal muscle mass and strength, and less adiposity.Nature communications · 2026Article
- Real-world evidence on nusinersen treatment of persons with SMA: a focused review.Journal of neuromuscular diseases · 2026Review
- Pharmacological intervention: Challenges and promising outcomes for fat loss and preservation of lean body mass in the treatment of overweight and type 2 diabetes.Diabetes, obesity & metabolism · 2026Review
- A type IV spinal muscular atrophy with gastrocnemius pseudohypertrophy caused by SMN1 deletion: a case report and literature review.BMC neurology · 2026Review
- Pharmacologic Treatments for the Preservation of Lean Body Mass During Weight Loss.Journal of clinical medicine · 2026Review
- Spinal muscular atrophy in the disease-modifying therapy era: successes, limitations and future directions.Frontiers in molecular medicine · 2026Review
- Spinal muscular atrophy: Biology, pathogenesis, and therapeutic advances.Therapeutic advances in neurological disorders · 2026Review
- Antibodies to watch in 2025.mAbs · 2025Review
- Emerging Role of Myostatin Inhibitors in the Management of Glucagon-Like Peptide-1-Associated Sarcopenia and Metabolic Disorders.Journal of bone metabolism · 2025Article
- Identification of p38 MAPK inhibition as a neuroprotective strategy for combinatorial SMA therapy.EMBO molecular medicine · 2025Article
- Glucocorticoid-Mediated Skeletal Muscle Atrophy: Molecular Mechanisms and Potential Therapeutic Targets.International journal of molecular sciences · 2025Review
Corrections and comments
- Erratum issued
- Erratum issued
Authors and funding
19 authors at 1 institution in 1 country.
Thomas O CrawfordFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0003-0916-2797
Basil T DarrasFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0002-1536-8145
John W DayFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0002-0086-9529
Sally Dunaway YoungFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0001-9322-0608
Tina DuongFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0003-3177-4722
Leslie L NelsonFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0003-3606-5757
Doreen BarrettFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0009-0004-7555-6422
Guochen SongFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0009-0009-9502-1093
Sanela BilicFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0009-0007-0181-7134
Shaun CoteFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0003-4596-9301
Mara SadanowiczFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0009-0005-0322-1110
Ryan IarrobinoFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0009-0007-1280-5649
Tiina J XuFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0009-0000-2093-0983
Janet O'NeilFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0009-0000-9209-868X
José RosselloFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0009-0002-2879-2380
Amy PlaceFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0003-4575-7632
Nathalie KerteszFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0001-6955-5934
George NomikosFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0000-0003-2846-1426
Yung ChyungFrom the Department of Neurology (T.O.C.), Johns Hopkins University, Baltimore, MD; Department of Neurology (B.T.D.), Boston Children's Hospital, Harvard Medical School, MA; Department of Neurology (J.W.D., S.D.Y., T.D.), Stanford University, Palo Alto, CA; Department of Physical Therapy (L.L.N.), University of Texas Southwestern Medical Center, Dallas; Scholar Rock, Inc. (D.B., G.S., S.C., M.S., R.I., T.J.X., J.O.N., J.R., A.P., N.K., G.N., Y.C.), Cambridge, MA; Vanadro, LLC (S.B.), Urbandale, IA; Tourmaline Bio, Inc. (R.I.), New York, NY; Pfizer, Inc. (A.P.), New York, NY; Harmony Biosciences (G.N.), Plymouth Meeting, PA; and Stealth BioTherapeutics (Y.C.), Needham, MA.ORCID 0009-0007-1548-8642
Boston Children's Hospital · US
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectivesCurrently approved therapies for spinal muscular atrophy (SMA) reverse the degenerative course, leading to better functional outcome, but they do not address the impairment arising from preexisting neurodegeneration. Apitegromab, an investigational, fully human monoclonal antibody, inhibits activation of myostatin (a negative regulator of skeletal muscle growth), thereby preserving muscle mass. The phase 2 TOPAZ trial assessed the safety and efficacy of apitegromab in individuals with later-onset type 2 and type 3 SMA.
methodsIn this study, designed to investigate potential meaningful combinations of eligibility and treatment regimen for future studies, participants aged 2-21 years received IV apitegromab infusions every 4 weeks for 12 months in 1 of 3 cohorts. Cohort 1 stratified ambulatory participants aged 5-21 years into 2 arms (apitegromab 20 mg/kg alone or in combination with nusinersen); cohort 2 evaluated apitegromab 20 mg/kg combined with nusinersen in nonambulatory participants aged 5-21 years; and cohort 3 blindly evaluated 2 randomized apitegromab doses (2 and 20 mg/kg) combined with nusinersen in younger participants ≥2 years of age. The primary efficacy measure was mean change from baseline using the Hammersmith Functional Motor Scale version appropriate for each cohort. Data were analyzed using a paired
resultsFifty-eight participants (mean age 9.4 years) were enrolled at 16 trial sites in the United States and Europe. Participants had been treated with nusinersen for a mean of 25.9 months before enrollment in any of the 3 trial cohorts. At month 12, the mean change from baseline in Hammersmith scale score was -0.3 points (95% CI -2.1 to 1.4) in cohort 1 (n = 23), 0.6 points (-1.4 to 2.7) in cohort 2 (n = 15), and in cohort 3 (n = 20), the mean scores were 5.3 (-1.5 to 12.2) and 7.1 (1.8 to 12.5) for the 2-mg/kg (n = 8) and 20-mg/kg (n = 9) arms, respectively. The 5 most frequently reported treatment-emergent adverse events were headache (24.1%), pyrexia (22.4%), upper respiratory tract infection (22.4%), cough (22.4%), and nasopharyngitis (20.7%). No deaths or serious adverse reactions were reported. DISCUSSION: Apitegromab led to improved motor function in participants with later-onset types 2 and 3 SMA. These results support a randomized, placebo-controlled phase 3 trial of apitegromab in participants with SMA. TRIAL REGISTRATION INFORMATION: This trial is registered with ClinicalTrials.gov (NCT03921528). CLASSIFICATION OF EVIDENCE: This study provides Class III evidence that apitegromab improves motor function in later-onset types 2 and 3 spinal muscular atrophy.
Indexed as
Antibodies, Monoclonal, HumanizedMuscular Atrophy, SpinalSpinal Muscular Atrophies of ChildhoodAntibodies, MonoclonalChildChild, PreschoolHumansInjections, SpinalAntibodies, MonoclonalAntibodies, Monoclonal, Humanizedapitegromab
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Read underepoch 390
Registered trials
NCT03921528ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.