Casimersen (AMONDYS 45^TM) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass developed by Sarepta therapeutics. It was approved by the Food and Drug Administration (FDA) in February 2021 to treat Duchenne muscular dystrophy (DMD) in patients whose DMD gene mutation is amenable to exon 45 skipping. Administered intravenously, casimersen binds to the pre-mRNA of the DMD gene to skip a mutated region of an exon, thereby producing an internally truncated yet functional dystrophin protein in DMD patients. This is essential in maintaining the structure of a myocyte membrane. While casimersen is currently continuing in phase III of clinical trials in various countries, it was granted approval by the FDA under the accelerated approval program due to its observed increase in dystrophin production. This article discusses the pathophysiology of DMD, summarizes available treatments thus far, and provides a full drug review of casimersen (AMONDYS 45^TM).

Background: Benzodiazepines (BZDs) place patients at a significant risk of falling. The current literature does not address if this risk is increased during initiation or dose escalations of BZDs. Objective: To determine if initiation or dose escalations of BZD regimens are associated with an increased risk of falls in hospitalized patients compared with patients maintained on their home dose or who had their dose decreased from baseline. Methods: This retrospective case-control study evaluated hospitalized patients aged 45 years or older who received a BZD. Patients who did not fall were collected in a 3:1 ratio to patients who fell. Comparisons were made between BZD regimens prior to admission and those 48 hours prior to the index date. The date of fall served as the index date for patients who fell, and the median time-to-fall served as the index date for all other patients. Results: A total of 132 patients were included in the study (33 falls and 99 without a fall). No significant differences were noted in demographics, baseline mobility, or past medical history. Patients who fell had a significantly longer median length of stay (15 vs 10 days; P = 0.025). Additionally, patients who fell were more likely to have had their BZD regimen initiated or dose escalated compared with patients who did not fall (63.6% vs 41.4%; P = 0.043). Conclusions: The risk of falling while on a BZD is increased on initiation and dose escalations. Hospitals should ensure judicious use of BZDs in inpatients to reduce the risk of falls.

The treatment of Cushing’s disease (CD) has been advanced well with the introduction of treatment options like transsphenoidal surgery, radiosurgery, bilateral adrenalectomy, and various classes of...

Objectives: Clostridium difficile infection (CDI) is the most common healthcare-associated infection in the United States. Clinical practice guidelines for the treatment of CDI were updated in 2010 by the Society for Healthcare Epidemiology of America and the Infectious Diseases Society of America. An institutional guideline for the classification and management of CDI in accordance with the 2010...

IntroductionMedication errors can lead to significant adverse events. Nearly 50% of medication errors occur during the prescription-writing stage of the medication use process, and effective interp...

The authors present a stereotypical case presentation of X-linked hypophosphatemia (XLH) and provide a review of the pathophysiology and related pharmacology of this condition, primarily focusing on the FDA-approved medication burosumab. XLH is a renal phosphate wasting disorder caused by loss of function mutations in the PHEX gene (phosphate-regulating gene with homologies to endopeptidases on the X chromosome). Typical biochemical findings include elevated serum levels of bioactive/intact fibroblast growth factor 23 (FGF23) which lead to (i) low serum phosphate levels, (ii) increased fractional excretion of phosphate, and (iii) inappropriately low or normal 1,25-dihydroxyvitamin D (1,25-vitD). XLH is the most common form of heritable rickets and short stature in patients with XLH is due to chronic hypophosphatemia. Additionally, patients with XLH experience joint pain and osteoarthritis from skeletal deformities, fractures, enthesopathy, spinal stenosis, and hearing loss. Historically, treatment for XLH was limited to oral phosphate supplementation, active vitamin D supplementation, and surgical intervention for cases of severe bowed legs. In 2018, the United States Food and Drug Administration (FDA) approved burosumab for the treatment of XLH and this medication has demonstrated substantial benefit compared with conventional therapy. Burosumab binds circulating intact FGF23 and blocks its biological effects in target tissues, resulting in increased serum inorganic phosphate (Pi) concentrations and increased conversion of inactive vitamin D to active 1,25-vitD.