Duchenne muscular dystrophy (DMD) is a debilitating and fatal genetic disease affecting 1/3500 boys globally, characterized by progressive muscle breakdown and eventual death with an average lifespan in the mid-late twenties. While no cure yet exists for DMD, gene and antisense therapies have been heavily explored in recent years to better treat this disease. Four antisense therapies have received conditional FDA approval, and many more exist in varying stages of clinical trials. These upcoming therapies often utilize novel drug chemistries to address limitations of existing therapies, and their development could herald the next generation of antisense therapy. This review article aims to summarize the current state of development for antisense-based therapies for the treatment of Duchenne muscular dystrophy, exploring candidates designed for both exon skipping and gene knockdown.

Immune-mediated necrotizing myopathy (IMNM) is a rare and severe disease that corresponds to a specific entity of idiopathic inflammatory myopathy. Patients with IMNM suffer from proximal muscle weakness, and present high levels of creatine kinase and necrotic myofibers. Anti-Signal Recognition Particle (SRP) and anti-3-hydroxy-3-methylglutaryl-coenzyme A reductase autoantibodies (HMGCR) have recently been identified in two thirds of patients with IMNM and are used as a hallmark of the disease. In this review, we provide a detailed description of these antibodies and the tests used to detect them in the serum of patients. Based on in vitro studies and mouse models of IMNM, we will discuss the role of autoantibodies in the pathogenesis of the disease. Finally, in the light of the latest knowledge, we will conclude with a review of recent therapeutic approaches.

In many Indian regions increased wastewater is both a threat and an opportunity. Efficient wastewater treatment and reuse schemes are needed in India to face impending water and fertiliser shortages. This study explores the application potential of wastewater fertigated Short Rotation Coppice systems (wfSRC) as a cost-efficient and promising solution for treating and reusing wastewater in a specific region (400 km2, 184 settlements) of Aligarh (UP), India. Based on real data from a local wfSRC pilot site using bamboo, willow, and poplar, we analysed the system’s treatment performance, nutrient recovery, carbon sequestration potential, land requirements, biomass production potential, and cost-benefit, under various scenarios. The results show that the pilot wfSRC system is efficiently treating 250 m3/day of domestic wastewater on 6,864 m2 of land, and serving 2,500 people. The land requirements for wfSRC systems vary depending on local conditions and user requirements, ranging from 2.75 – 25.7 m2/PE which equates to a required land area in the whole study region of between 108 and 1006 ha in 2036. This would produce up to 100 DM t/ha/yr of valuable biomass. Early local stakeholder involvement and monitoring of pollutants are recommended as priorities during the planning process for large-scale implementation of wfSRC systems in India.