Innovative Technology, Clinical Trials and the Subjective Evaluation of Patients: The Cyborg-type Robot HAL and the Treatment of Functional Regeneration in Patients with Rare Incurable Neuromuscular Diseases in Japan

2021 ◽  
pp. 281-310
Author(s):  
Takashi Nakajima
Keyword(s):  
Clinical Trials ◽  
Subjective Evaluation ◽  
Neuromuscular Diseases ◽  
Innovative Technology ◽  
Functional Regeneration

57P Effective recruitment strategies for clinical trials in rare neuromuscular diseases

1994 ◽  
Vol 15 (3) ◽  
pp. 109
Author(s):  
Barbara Herr ◽  
Lynn Cos ◽  
Cynthia Palenski ◽  
Shree Pandya ◽  
Norine Stirpe
Keyword(s):  
Clinical Trials ◽  
Neuromuscular Diseases ◽  
Recruitment Strategies

scholarly journals Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases

2021 ◽  
Vol 12 ◽  
Author(s):  
Tina Duong ◽  
Kristin J. Krosschell ◽  
Meredith K. James ◽  
Leslie Nelson ◽  
Lindsay N. Alfano ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Clinical Outcome ◽  
High Reliability ◽  
Neuromuscular Diseases ◽  
Reliability And Validity ◽  
Standards Of Care ◽  
Roles And Responsibilities ◽  
Key Stakeholders ◽  
Critical Components ◽  
Clinical Outcome Assessments

Critical components of successful evaluation of clinical outcome assessments (COAs) in multisite clinical trials and clinical practice are standardized training, administration, and documented reliability of scoring. Experiences of evaluators, alongside patient differences from regional standards of care, may contribute to heterogeneity in clinical center’s expertise. Achieving low variability and high reliability of COA is fundamental to clinical research and to give confidence in our ability to draw rational, interpretable conclusions from the data collected. The objective of this manuscript is to provide a framework to guide the learning process for COAs for use in clinics and clinical trials to maximize reliability and validity of COAs in neuromuscular disease (NMD). This is a consensus-based guideline with contributions from fourteen leading experts in clinical outcomes and the field of clinical outcome training in NMD. This framework should guide reliable and valid assessments in NMD specialty clinics and clinical trials. This consensus aims to expedite study start up with a progressive training pathway ranging from research naïve to highly experienced clinical evaluators. This document includes recommendations for education guidelines and roles and responsibilities of key stakeholders in COA assessment and implementation to ensure quality and consistency of outcome administration across different settings.

scholarly journals Wearable sensor-based data analysis for neurological disease symptoms evaluation utilising quantitative approach.

2018 ◽  
Vol 210 ◽  
pp. 05015 ◽  
Author(s):  
Mariusz Chmielewski ◽  
Michał Nowotarski
Keyword(s):  
Clinical Trials ◽  
Neurological Diseases ◽  
Subjective Evaluation ◽  
Wearable Sensors ◽  
Monitoring And Evaluation ◽  
Quantitative Approach ◽  
Health State ◽  
State Evaluation ◽  
Novel Approach ◽  
Mental Slowness

The paper describes implementation of an analytical method and conclusions of novel approach to clinical trials monitoring and evaluation. Based on clinical trials observations a set of requirements for validating symptoms of neurological diseases have been formulated, concentrating on the ones which can be registered using wearable sensors. The constructed tool utilizes conventional surveying methods supplemented with biomedical sensor for neurological symptoms recognition and intensity evaluation. Developed mobile system is aimed at clinical trials assistance utilising sensor-based state evaluation. Such quantitative approach is a supplement for patient’s subjective evaluation of health state. This work is a discussion on pros and cons of such process composition and its supplementation with technology. Existing methodology relies on health state evaluation based on iteratively answered questionnaires, which in our understanding cannot be fully controlled and reliable. Utilisation of actigraphy and electromyography provides efficient means of some gestures recognition but most of all PD tremor identification and evaluation of their intensity, therefore can be used for ON/OFF state and dyskinesia identification and evaluation. In order to recognise specific states for PD patients (tremors, bradykinesias, rigidity, mental slowness, etc.) a set of additional techniques have been designed and implemented.

scholarly journals Current Therapeutic Approaches in FSHD

2020 ◽  
pp. 1-11
Author(s):  
Leo H. Wang ◽  
Rabi Tawil
Keyword(s):  
Skeletal Muscle ◽  
Clinical Trials ◽  
Neuromuscular Diseases ◽  
Underlying Disease ◽  
Facioscapulohumeral Muscular Dystrophy ◽  
Muscular Dystrophies ◽  
Therapeutic Approaches ◽  
Disease Mechanism ◽  
First Time ◽  
Targeted Approach

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common muscular dystrophies. Over the last decade, a consensus was reached regarding the underlying cause of FSHD allowing—for the first time—a targeted approach to treatment. FSHD is the result of a toxic gain-of-function from de-repression of the DUX4 gene, a gene not normally expressed in skeletal muscle. With a clear therapeutic target, there is increasing interest in drug development for FSHD, an interest buoyed by the recent therapeutic successes in other neuromuscular diseases. Herein, we review the underlying disease mechanism, potential therapeutic approaches as well as the state of trial readiness in the planning and execution of future clinical trials in FSHD.

Therapies for RYR1-Related Myopathies: Where We Stand and the Perspectives

2021 ◽  
Vol 27 ◽  
Author(s):  
Mathilde Beaufils ◽  
Lauriane Travard ◽  
John Rendu ◽  
Isabelle Marty
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Rare Diseases ◽  
Clinical Assessment ◽  
Neuromuscular Diseases ◽  
Ryr1 Gene ◽  
Therapeutic Development ◽  
Therapy Strategies

: RyR1-related myopathies are a family of genetic neuromuscular diseases due to mutations in the RYR1 gene. No treatment exists for any of these myopathies today, which could change in the coming years with the growing number of studies dedicated to the pre-clinical assessment of various approaches, from pharmacological to gene therapy strategies, using the numerous models developed up to now. In addition, the first clinical trials for these rare diseases have just been completed or are being launched. We review the most recent results obtained for the treatment of RyR1-related myopathies, and, in view of the progress in therapeutic development for other myopathies, we discuss the possible future therapeutic perspectives for RyR1-related myopathies.

scholarly journals Secure and Scalable mHealth Data Management Using Blockchain Combined With Client Hashchain: System Design and Validation (Preprint)

2019 ◽  
Author(s):  
Tomomitsu Motohashi ◽  
Tomonobu Hirano ◽  
Kosuke Okumura ◽  
Makiko Kashiyama ◽  
Daisuke Ichikawa ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Data Management ◽  
Mobile Devices ◽  
Medical Practice ◽  
Chain Structure ◽  
Medical Data ◽  
Third Party ◽  
Innovative Technology ◽  
Blockchain Technology ◽  
Network Fault

BACKGROUND Blockchain is emerging as an innovative technology for secure data management in many areas, including medical practice. A distributed blockchain network is tolerant against network fault, and the registered data are resistant to tampering and revision. The technology has a high affinity with digital medicine like mobile health (mHealth) and provides reliability to the medical data without labor-intensive third-party contributions. On the other hand, the reliability of the medical data is not insured before registration to the blockchain network. Furthermore, there are issues with regard to how the clients' mobile devices should be dealt with and authenticated in the blockchain network in order to avoid impersonation. OBJECTIVE The aim of the study was to design and validate an mHealth system that enables the compatibility of the security and scalability of the medical data using blockchain technology. METHODS We designed an mHealth system that sends medical data to the blockchain network via relay servers. The architecture provides scalability and convenience of operation of the system. In order to ensure the reliability of the data from clients' mobile devices, hash values with chain structure (client hashchain) were calculated in the clients' devices and the results were registered on the blockchain network. RESULTS The system was applied and deployed in mHealth for insomnia treatment. Clinical trials for mHealth were conducted with insomnia patients. Medical data of the recruited patients were successfully registered with the blockchain network via relay servers along with the hashchain calculated on the clients' mobile devices. The correctness of the data was validated by identifying illegal data, which were made by simulating fraudulent access. CONCLUSIONS Our proposed mHealth system, blockchain combined with client hashchain, ensures compatibility of security and scalability in the data management of mHealth medical practice. CLINICALTRIAL UMIN Clinical Trials Registry UMIN000032951; https://upload.umin.ac.jp/cgi-open- bin/ctr_e/ctr_view.cgi?recptno=R000037564 (Archived by WebCite at http://www.webcitation.org/78HP5iFIw)

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