Efficacy and safety of Nusinersen among children with spinal muscular atrophy from North India: A prospective cohort study (NICE-SMA study).

Background: Intra-thecal Nusinersen has been approved for the treatment of Spinal muscular atrophy (SMA). Limited data is available regarding the efficacy and safety of Nusinersen in children with SMA type 2 and 3 from North India.

Objective: To study the efficacy and safety of Nusinersen among children with SMA type 2 and 3 from North India compared to standard of care (SOC) over 12 months.

Development and Economic Evaluation of a Patient-Centered Care Model for Children With Duchenne Muscular Dystrophy: Protocol for a Quasi-Experimental Study.

Background: Duchenne muscular dystrophy (DMD) is a rare progressive muscular disease that primarily affects boys. A lack of comprehensive care for patients living with DMD is directly associated with a compromised quality of life (QoL) for those affected and their caregivers. This disease also has a huge economic impact on families as its treatment requires substantial direct, indirect, and informal care costs.

Deflazacort dose optimization and safety evaluation in Duchenne muscular dystrophy (DOSE): A randomized, double-blind non-inferiority trial.

Background: US food and drug administration has recently approved deflazacort for Duchenne muscular dystrophy (DMD) and recommended the dosage of 0.9 mg/kg/d for patients aged ≥5years. However, data assessing the minimal efficacious dose and need of dose-titration based on age or disease severity is limited.

Comparative performance of verbal autopsy methods in identifying causes of adult mortality: A case study in India.

Background & Objectives: Cause of death assignment from verbal autopsy (VA) questionnaires is conventionally accomplished through physician review. However, since recently, computer softwares have been developed to assign the cause of death. The present study evaluated the performance of computer software in assigning the cause of death from the VA, as compared to physician review.

The Expanding Spectrum of Dystrophinopathies: HyperCKemia to Manifest Female Carriers.

Background: X-linked dystrophinopathies have a wide spectrum of manifestation. The most common forms are severe Duchenne muscular dystrophy (DMD) and Becker's muscular dystrophy (BMD). However, less common manifestations are isolated cardiomyopathy, myalgia, cramps, rhabdomyolysis, hyperCKemia, and manifest female carriers.

Bone density and bone health alteration in boys with Duchenne Muscular Dystrophy: a prospective observational study.

Objectives: Boys with Duchenne Muscular Dystrophy (DMD) are at increased risk for compromised bone health, manifesting as low-impact trauma long bone fractures and vertebral compression fractures.

Methods: In a prospective observational study, we studied bone health parameters in North Indian boys with DMD. We consecutively enrolled ambulatory boys with DMD on glucocorticoid therapy.

Social autopsy for identifying causes of adult mortality.

Verbal autopsy methods have been developed to determine medical causes of deathforprioritizing disease control programs. Additional information on social causesmay facilitate designing of more appropriate prevention strategies. Use of social autopsy in investigations of causes of adult deaths has been limited.