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.

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).

Transcutaneous Electrical Nerve Stimulation in Management of Neurogenic bladder Secondary to Spina Bifida.

Aims: To assess the role of transcutaneous electrical nerve stimulation (TENS), alone or in combination with anticholinergic drugs in the management of neurogenic bladder (NB) in spina bifida (SB).

Materials And Methods: All the consecutive patients, visiting outpatient clinic between July 2017 and December 2018, who were toilet trained and at least 1 year post-SB surgery with clinical and/or urodynamic evidence of NB, were included in the study. Out of 65 patients, 40 fulfilled the inclusion criteria and were randomised into: group A (ten patients, placebo TENS with anticholinergic agents), Group B (14 patients, TENS therapy with placebo medications) and Group C (16 patients, TENS therapy with anticholinergic medications).

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.

Pilot study of rapamycin in patients with hormone-refractory prostate cancer.

Background: Currently, no second-line treatment exists for hormonerefractory prostate cancer (HRPC) cases that fail docetaxel regimens. Rapamycin, an immunosuppressive macrolide, inhibits metastatic prostate tumor growth and angiogenesis in in vivo mouse models. This pilot study assessed the antitumor activity, safety, and toxicity of rapamycin in patients with HRPC.

Vaccination of prostate cancer patients with modified vaccinia ankara delivering the tumor antigen 5T4 (TroVax): a phase 2 trial.

The attenuated vaccinia virus, modified vaccinia Ankara, has been engineered to deliver the tumor antigen 5T4 (TroVax). TroVax has been evaluated in an open-label phase 2 trial in hormone refractory prostate cancer patients in which the vaccine was administered either alone or in combination with granulocyte macrophage-colony stimulating factor (GM-CSF). The comparative safety and immunologic and clinical efficacy of TroVax alone or in combination with GM-CSF was determined.