Evidence map›Paper›PMID 38318125›Full record

ArticleEClinicalMedicine2024

Rycal S48168 (ARM210) for

Joshua J Todd, Tokunbor A Lawal, Irene C Chrismer, Angela Kokkinis, Christopher Grunseich, Minal S Jain, Melissa R Waite, Victoria Biancavilla, Shavonne Pocock, Kia Brooks and 14 more

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in EClinicalMedicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04141670 (Safety and Tolerability of S 48168), which is not on this map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

NCT04141670 phase1completednot on this map

Safety and Tolerability of S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)

TypeinterventionalSponsorRyCarma Therapeutics, Inc.Ran2020 to 2023Enrolled7ConditionsRYR-1 MyopathyArmsS48168
3 · Its place in the literature

Who cites it

14 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. Review
  5. Article
  6. Review
  7. Phosphorylation of RYR1 at SerScience signaling · 2025
    Article
  8. Article
  9. Review
  10. NADSkeletal muscle · 2025
    Article
  11. Article
  12. Effects of aging on calcium channels in skeletal muscle.Frontiers in molecular biosciences · 2025
    Review
  13. Review
  14. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

24 authors.

Joshua J ToddNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20814, USA.
Tokunbor A LawalNational Institute of Nursing Research, National Institutes of Health, Bethesda, MD 20814, USA.
Irene C ChrismerNational Institute of Nursing Research, National Institutes of Health, Bethesda, MD 20814, USA.
Angela KokkinisNeurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20814, USA.
Christopher GrunseichNeurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20814, USA.
Minal S JainMark O. Hatfield Clinical Research Center, Rehabilitation Medicine Department, National Institutes of Health, Bethesda, MD 20814, USA.
Melissa R WaiteMark O. Hatfield Clinical Research Center, Rehabilitation Medicine Department, National Institutes of Health, Bethesda, MD 20814, USA.
Victoria BiancavillaMark O. Hatfield Clinical Research Center, Rehabilitation Medicine Department, National Institutes of Health, Bethesda, MD 20814, USA.
Shavonne PocockNational Institute of Nursing Research, National Institutes of Health, Bethesda, MD 20814, USA.
Kia BrooksNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20814, USA.
Christopher J MendozaNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20814, USA.
Gina NoratoClinical Trials Unit, National Institute of Neurological Disorders and Stroke, Bethesda, MD, USA.
Ken CheungMailman School of Public Health, Columbia University, NY 10032, USA.
Willa RiekhofNational Institute of Nursing Research, National Institutes of Health, Bethesda, MD 20814, USA.
Pooja VarmaNational Institute of Nursing Research, National Institutes of Health, Bethesda, MD 20814, USA.
Claudia Colina-PriscoSection of Sensory Science and Metabolism, National Institute on Alcohol Abuse and Alcoholism, National Institutes of Health, Bethesda, MD 20814, USA.
Magalie Emile-BackerNational Institute of Nursing Research, National Institutes of Health, Bethesda, MD 20814, USA.
Katherine G MeilleurNational Institute of Nursing Research, National Institutes of Health, Bethesda, MD 20814, USA.
Andrew R MarksDepartment of Physiology and Cellular Biophysics, Columbia University Vagelos College of Physicians and Surgeons, New York, NY 10032, USA.
Yael WebbARMGO Pharma, Inc, Ardsley, NY 10591, USA.
Eugene E MarcantonioARMGO Pharma, Inc, Ardsley, NY 10591, USA.
A Reghan FoleyNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20814, USA.
Carsten G BönnemannNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20814, USA.
Payam MohasselNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20814, USA.

Funding

Molecular and Clinical Manifestations of Matrix and Aggregate MyopathiesZIANS003129 · NINDS · NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE · PI BÖNNEMANN, CARSTEN · 2011 to 2025
$52.8M
Functional characterization of genetic variants of uncertain significance in genes associated with excitation-contraction coupling disordersZIANR000040 · NINR · NATIONAL INSTITUTE OF NURSING RESEARCH · PI LAWAL, TOKUNBOR · 2023 to 2025
$895k
The role of TGF beta pathway dysregulation in pathogenesis of collagen VI-related muscular dystrophyK22NS104135 · NINDS · JOHNS HOPKINS UNIVERSITY · PI MOHASSEL, PAYAM · 2022 to 2024
$666k
NINDS NIH HHS K22 NS104135
6 · The paper itself

Abstract

Background: Methods: Participants received 120 mg (n = 3) or 200 mg (n = 4) S48168 (ARM210) daily for 29 days. The primary endpoint was safety and tolerability. Exploratory endpoints included S48168 (ARM210) pharmacokinetics (PK), target engagement, motor function measure (MFM)-32, hand grip and pinch strength, timed functional tests, PROMIS fatigue scale, semi-quantitative physical exam strength measurements, and oxidative stress biomarkers. The trial was registered with clinicaltrials.gov (NCT04141670) and was conducted at the National Institutes of Health Clinical Center between October 28, 2019 and December 12, 2021. Findings: S48168 (ARM210) was well-tolerated, did not cause any serious adverse events, and exhibited a dose-dependent PK profile. Three of four participants who received the 200 mg/day dose reported improvements in PROMIS-fatigue at 28 days post-dosing, and also demonstrated improved proximal muscle strength on physical examination. Interpretation: S48168 (ARM210) demonstrated favorable safety, tolerability, and PK, in Funding: NINDS and NINR Intramural Research Programs, NIH Clinical Center Bench to Bedside Award (2017-551673), ARMGO Pharma Inc., and its development partner Les Laboratoires Servier.

Indexed as

Central core diseaseCongenital myopathies

Identifiers

PMID38318125
PMCPMC10839573

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

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.