Critical Care

Spinal Muscular Atrophy (SMA) is a genetic disorder causing muscle weakness and atrophy due to the degeneration of alpha motor neurons in the spinal cord. SMA is a major cause of hereditary mortality globally, with carriers estimated to be 1/40–1/60. Assistive equipment include, adaptive strollers, wheelchairs, and support devices. Target therapies like Nusinersen, Risidiplam, and Onasemnogene abeparvovec target the underlying disease mechanism and may prevent or slow SMA progression. Risdiplam compensates for the loss of SMN2 function in SMA patients, while Onasemnogene abeparvovec is a gene therapy that delivers a functional copy of the human survival motor neuron gene to patients, showing significant improvement in developmental motor milestones. Nusinersen is an antisense oligonucleotide (ASO) used to treat 5q Spinal Muscular Atrophy (5q SMA). It alters SMN2 gene splicing to increase SMN protein synthesis, correcting the disease’s underlying cause. Nusinersen helps produce full-length (100%) SMN protein essential for motor neuron function. Palliative, supportive, and rehabilitative treatment for SMA includes orthopaedic care, dietary assistance, end-of-life care, and pulmonary management, with severity varying based on disease type. The numerous therapy approaches to treat and assist people with SMA are the main topic of this blog. Read More

Cardiovascular disease continues to be a major concern, and the electrocardiogram (ECG) is a proven non-invasive technique for detecting cardiac issues. Traditional diagnosis, on the other hand, is based on an individual patient’s medical history and clinical examinations, which are ineffective owing to diverse data. By analyzing the electrical activity of the heart, AI is being used to identify prognostic arrhythmias such as atrial fibrillation. Deep convolutional neural networks (CNNs) are the basic building blocks of machine learning algorithms used in cardiovascular medicine to analyze ECG data. The adoption and use of AI-based diagnostic tools in clinical settings, however, may be limited by issues with interpretability and openness, such as evaluating models’ performance across datasets, processing power consumption, privacy and security concerns, imbalanced and limited datasets, and lack of clear guidelines for CNNs. Nevertheless, these technologies offer standardization, continuous, real-time monitoring, and more accurate interpretation—all of which have the potential to improve patient outcomes. This blog provides an overview of AI technologies applied and the challenges associated with the ECG in the diagnosis of cardiovascular diseases. Read More

Constant emergence of new gene therapies as well as refinement of the existing ones changes the global landscape of the cell and gene therapies clinical trials, where the US, China, and Europe are leading in respect of the number of trials conducted. As per Global Data, China showed 15% faster growth in cell and gene therapy clinical trials making the Asia-Pacific region contributes for one-third of the trial activities. As a result, the Asia Pacific region is witnessing 50% faster growth than the rest of the world (ROW). Asia Pacific region leads globally in terms of CAR-T cell gene therapy clinical trials for the time period 2015-2022 since China alone conducted ~60% of all CAR-T trials. Till April 2022, there are 19 approved gene therapies, 17 RNA-approved therapies while 56 non-genetically modified approved cell therapies (Figure 1). Details of the approved location of the clinical trials of gene therapies and RNA therapies drug product are provided in Table No.1 and Table No. 2 respectively, which presents a bird’s-eye view of the landscape of the clinical trials of the approved gene and RNA therapies. Read More

In the journey of our Cancer Immunotherapy blog series, let us introduce CAR-T cell therapy, another milestone in recent years in the field of immunotherapy that has revolutionized the modern medicine. Chimeric Antigen Receptor (CAR) T cell therapy utilizes T-cells, a type of white blood cell (immune cells), to fight cancer by engineering them ex vivo prior to infusing back into the patient. These CAR T-cells can specifically find and destroy cancerous cells. CAR T-cell therapy is a type of cell-based gene therapy or Adoptive Cell Therapy (ACT) as it involves gene alteration of T-cells that enables them to attack specific cancer cells. Read More