日本語 EN 中文

2023.12.27

Erythromycin for myotonic dystrophy type 1: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

Overview


A research group from Yamaguchi University and Osaka University has confirmed for the first time worldwide the efficacy of the existing drug erythromycin for myotonic dystrophy. This intractable disease, caused by splicing abnormalities due to abnormal RNA, had no fundamental treatment.
The research group conducted a multicenter clinical trial, confirming erythromycin’s safety. Furthermore, they demonstrated its efficacy in improving splicing abnormalities, the underlying cause of the disease. This finding holds promise for erythromycin becoming the world’s first treatment for myotonic dystrophy.
These research results were published in the UK-based international academic journal “eClinicalMedicine.”

RICeD Member
  • Division of Advanced Genome Editing Therapy
  • 　　　Masayuki Nakamori (Neurology)
Paper Information
  • Title: Erythromycin for myotonic dystrophy type 1: a multicenter, randomized, double-blind, placebo-controlled, phase 2 trial
  • (Treatment of myotonic dystrophy with erythromycin: a multicenter, randomized, placebo-controlled, double-blind comparative trial)
  • Authors: Nakamori M1,2, Nakatani D3, Sato T4, Hasuike Y2, Kon S5, Saito T6, Nakamura H7, Takahashi MP8, Hida E4, Komaki H7, Matsumura T6, Takada H5, and Mochizuki H2
  • Affiliation:
  • 1. Yamaguchi University Graduate School of Medicine, Department of Clinical Neurology
  • 2. Osaka University Graduate School of Medicine, Department of Neurology
  • 3. Osaka University Hospital, Department of Future Medical Development
  • 4. Osaka University Graduate School of Medicine, Joint Research Program in Medical Data Science
  • 5. NHO Aomori Hospital
    6. NHO Osaka Toneyama Medical Center
  • 7. National Center of Neurology and Psychiatry, Translational Medical Center
  • 8. Osaka University Graduate School of Medicine, Department of Clinical Neurophysiology
  • Publication：eClinicalMedicine (2024)
  • D O I：https://doi.org/10.1016/j.eclinm.2023.102390
Contact
  • ＜Research Inquiries＞
  • Neurology, Yamaguchi University
  • Professor Masayuki Nakamori
  • TEL: 0836-22-2719/2713
  • E-mail: mnakamor@yamaguchi-u.ac.jp
  • ＜Public Relations＞
  • Yamaguchi University Research Institute for Cell Design Medical Science (Life Science Support Section, Academic Research Division)
  • Tel: 0836-85-3065
  • E-mail: sh088@yamaguchi-u.ac.jp

Back