Commonly available outcome measures for use in Indian boys with Duchenne muscular dystrophy

Area of Science:

• Genetics and Molecular Biology
• Neurology
• Clinical Trials

Background:

• Duchenne muscular dystrophy (DMD) is a severe X-linked recessive disorder.
• Currently, DMD remains an incurable condition, necessitating ongoing research and clinical trials.
• Accurate assessment of disease progression is vital for evaluating therapeutic interventions.

Purpose of the Study:

• To review and identify sensitive and reliable outcome measures for Duchenne muscular dystrophy patients in India.
• To provide a resource for researchers and clinicians involved in DMD clinical trials in the Indian context.

Main Methods:

• Literature review of existing outcome measures used in Duchenne muscular dystrophy studies.
• Focus on measures applicable and validated for the Indian population.
• Analysis of sensitivity, reliability, and clinical relevance of identified outcome measures.

Main Results:

• Several potential outcome measures were identified, including functional assessments, muscle strength tests, and biomarkers.
• The applicability and feasibility of these measures in the Indian healthcare setting were considered.
• The review highlights the need for standardized protocols for outcome measure implementation.

Conclusions:

• Standardized and validated outcome measures are essential for effective Duchenne muscular dystrophy clinical trial management in India.
• The selection of appropriate outcome measures can significantly impact the interpretation of treatment efficacy.
• Further research may be needed to validate specific measures for the Indian DMD population.