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.

Synthesis of imidazole-fused nitrogen-bridgehead heterocycles catalysed by lipase and their antifungal and antimicrobial bioactivity.

An effective approach for selective C-N bond formation for synthesising imidazo[1,2-] pyridine-based heterocycles using porcine pancreatic lipase (PPL) as a biocatalyst has been devised. Under moderate conditions, a series of imidazo[1,2-]pyridine-based heterocycle derivatives were synthesised with remarkable selectivity in good-to-excellent yields (89-95%). Further, the antimicrobial and antifungal activities of derivatives 3ha, 3ka, 3fa, 3hc, and 3eb were observed, and they were found to be biologically active in antimicrobial susceptibility tests for Gram-positive bacteria ( and ), Gram-negative bacteria ( and ) and fungal strains ( and ).

Efficacy of Teleconsultation-Based Rehabilitation in Children with Landry-Guillain-Barré Syndrome: An Open Label Randomized Controlled Trial (Tele Rehab-LGBS Trial).

Objectives: To evaluate the feasibility and efficacy of teleconsultation-based rehabilitation in children with Landry-Guillain-Barré syndrome (LGBS), measured with Medical Research Council (MRC) sum score and Hughes score.

Methods: A pragmatic, prospective, parallel open label randomized controlled trial was conducted among a total of 50 children with LGBS. The children were randomized using computer generated block randomization into 2 groups (25 in each group): Standard of care (Group A) and teleconsultation-based rehabilitation (Group B).

Electro-organic synthesis of isatins and hydrazones through C-N cross-coupling and C(sp)-H/C(sp)-H functionalization.

An efficient and unique approach to synthesize isatin (indole-2,3-dione) from 2-aminoacetophenone under electrochemical conditions supported by I-DMSO through C-N cross-coupling and C(sp)-H/C(sp)-H functionalization is presented. This synthetic method spans a wide range of substituted 2-aminoacetophenone substrates. The use of iodine as a promoter and shorter reaction times produced good to very good yields of isatin derivatives, which is a significant improvement over the reaction in a batch process.

Electro-organic green synthesis of dicyano-2-(2-oxoindolin-3-ylidene) malononitriles using molecular iodine as catalyst.

The first electrochemical molecular iodine promoted, domino reactions for the green synthesis of biologically relevant dicyano 2-(2-oxoindolin-3-ylidene) malononitriles (11 examples, up to 94% yield) from readily available isatin derivatives, malononitrile, and iodine at room temperature have been presented. This synthesis method showed tolerance towards various EDG and EWG and was completed in a short reaction time at the constant low current density of 5 mA cm in the low redox potential range of -0.14 to 0.

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.

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.

Efficient one-pot synthesis of substituted diphenyl 1, 3-thiazole through multicomponent reaction by using green and efficient Iron-catalyst via Cross-Dehydrogenative Coupling(CDC).

A clean and efficient, multi-component strategy for the synthesis of biologically important trisubstituted thiazole via the reaction of readily available barbituric acid, acetophenone, and aryl thioamides is reported in the presence of FeCl.6HO / O(Air) in DMF solvent. The advantages of the present methodology include a one-pot reaction, environment-friendly approach, cost-effectiveness, broad substrate scope, operational simplicity, short reaction time, easy workup procedure, and high yields.

Ag-Cu Bimetallic Nanoparticles as Efficient Oxygen Reduction Reaction Electrocatalysts in Alkaline Media.

The alkaline medium oxygen reduction reaction (ORR) activities of Ag-Cu bimetallic nanoparticles (BNPs), consisting of neighboring Ag and Cu domains, were studied and compared with those of pure Ag and Cu nanoparticles prepared by the same polyol route. Three variations of Ag-Cu BNPs viz. Ag-Cu (4:1), Ag-Cu (2:1), Ag-Cu (1:1) BNPs were considered.

Enhanced electron transfer mediated detection of hydrogen peroxide using a silver nanoparticle-reduced graphene oxide-polyaniline fabricated electrochemical sensor.

The current study aims at the development of an electrochemical sensor based on a silver nanoparticle-reduced graphene oxide-polyaniline (AgNPs-rGO-PANI) nanocomposite for the sensitive and selective detection of hydrogen peroxide (HO). The nanocomposite was fabricated by simple synthesis of PANI at the surface of rGO sheet which was followed by stirring with AEC biosynthesized AgNPs to form a nanocomposite. The AgNPs, GO, rGO, PANI, rGO-PANI, and AgNPs-rGO-PANI nanocomposite and their interaction were studied by UV-vis, FTIR, XRD, SEM, EDX and XPS analysis.