Have you completed patient registration (Remudy)?

Our patient association is prioritizing patient registration (Remudy) as our top priority.

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Patient registration (Remudy) is conducted by the Neurological and Muscular Disease Patient Registration Center, and registration for FSHD patients began in September 2020.

 

The benefits of patient registration (Remudy) are summarized below.

• Through the patient registration (Remudy) application, the latest diagnostic standards required for clinical trial participation and treatment selection will be conducted. (Note 1) (Note 2)

• By updating patients' physical condition data annually, it is possible to objectively assess their medical health status.

• Patient registration (Remudy) is linked to the international patient registry (TREAT-NMD), which can serve as a foundation for participating in international clinical trials.

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Note: While participation in the international clinical trial for Rosumapimod is not currently possible, it is anticipated that as the number of registrants increases significantly, opportunities for participation in international clinical trials may arise in the future. Participation in international clinical trials could enable simultaneous international approval, contributing to the resolution of medical drug lag (Note 3).

 

(Note 1) Patients may decline to participate in clinical trials if they determine that there are risks involved.

(Note 2) Patients may participate in clinical trials through their attending physician or other means without registering with Remudy. Clinical trials are planned and conducted by pharmaceutical companies, and the method of recruitment is determined by the company.

(Note 3) The issue of significant time required for a drug already approved overseas to obtain approval in Japan.

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◇Registration Method

The patient registration form (Remudy) can be downloaded from the “Document Download” section of the website. ↓

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Neuromuscular Disease Patient Registration Remudy Facial-scapular-brachial type muscular dystrophy

https://remudy.ncnp.go.jp/fshd/

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• Consent Form (for the patient)

• Consent Form (for the attending physician)

• FSHD Patient Registration Form

Print these three documents and request your attending physician's cooperation.

If you do not have a printer, they can be mailed to you.

(Postage and other costs for mailing the registration forms are the responsibility of the applicant.)

 

If you have any questions, please contact the Remudy Patient Registration Office.

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187-8551 Tokyo, Kodaira City, Ogawa Higashi-cho 4-1-1

National Center of Neurology and Psychiatry

Neuromuscular Disease Patient Registration Center Remudy (Remudy) Office

 

Phone: 042-346-2309

 

◇Genetic Diagnosis

Patient registration (Remudy) is available for individuals who have been diagnosed through genetic testing (Note 4).

Those who underwent genetic testing prior to June 2018 may require additional testing.

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(Note 4) The genetic diagnostic methods for FSHD have evolved over time. Since the mechanism of FSHD onset has not yet been definitively established, a true definitive diagnostic method has not yet been established, and the diagnosis is based on the current diagnostic criteria.

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Previously, FSHD1 was diagnosed based on the result that the DNA in the D4Z4 region of chromosome 4 was shorter (with six or fewer repeats). However, with recent advances in genetic analysis, it has been discovered that even in cases where the DNA is shorter (with six or fewer repeats), there are rare cases where the individual is not FSHD1 based on haplotype analysis results.

Therefore, to definitively diagnose FSHD1, additional detailed testing called haplotype analysis is required. (Over 90% of FSHD patients are FSHD1, with the remainder being FSHD2.)

The source of the diagram below is from NCNP.