Approvals in 2017: gene therapies and site-agnostic indications

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

Certain organic substances change color in dilute solution when the hydronium ion concentration reaches a particular value. For example, phenolphthalein is a colorless substance in any aqueous solution with a hydronium ion concentration greater than 5.0 × 10−9 M (pH < 8.3). In more basic solutions where the hydronium ion concentration is less than 5.0 × 10−9 M (pH > 8.3), it is red or pink. Substances such as phenolphthalein, which can be used to determine the pH of a solution, are...

Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...

Mechanical ventilation is a life-saving technique for managing acute respiratory failure and other respiratory complications. The process involves using a machine known as a ventilator to supply oxygen to the lungs and assist in removing carbon dioxide. It serves as a bridge to long-term mechanical ventilation or a temporary measure until ventilatory support is discontinued. The ventilator can maintain this function for a prolonged period, providing critical support for patients until they can...

Respiratory assessment is a cornerstone of nursing assessments, crucial for the early detection of patient deterioration. This evaluation transcends routine procedures, representing a critical skill nurses must master to ensure optimal patient care.
Objectives and Importance:
The primary goal of respiratory assessment is to evaluate patients at early risk of clinical deterioration. Since respiratory distress often precedes other signs of declining health, breathing patterns and sounds become a...

Group therapy is a sociocultural approach to psychological treatment, where individuals with shared psychological challenges come together under the guidance of a mental health professional. This therapeutic modality offers unique opportunities for individuals to connect, share, and grow within the context of a supportive group. By fostering mutual understanding and collaboration, group therapy can address a range of psychological concerns effectively, often complementing or surpassing the...