Daniel J. Berlau

Duchenne muscular dystrophy (DMD) is a severe X-linked disorder caused by mutations in the dystrophin gene, resulting in progressive skeletal muscle degeneration, loss of ambulation, and eventual cardiopulmonary failure. Although survival has improved, DMD remains incurable and associated with substantial morbidity and premature mortality. A comprehensive literature search was conducted to examine current and prospective pharmacotherapeutics for the treatment of DMD. This review summarizes FDA-approved therapies for DMD, emphasizing mechanisms of action, clinical efficacy, and safety. Corticosteroids remain the standard of care, with newer agents, such as vamorolone offering alternative safety profiles. Mutation-specific exon-skipping therapies and adeno-associated virus-mediated microdystrophin gene transfer have expanded treatment options but show variable functional benefit and safety concerns. The recent approval of the histone deacetylase inhibitor givinostat is also discussed. Emerging strategies include next-generation gene editing, utrophin upregulation, and diverse cell-based therapies, alongside evolving approaches to cardiac management. Recent therapeutic expansion has been driven in part by accelerated regulatory pathways relying on surrogate endpoints. While these advances provide important access, long-term efficacy, and safety data remain limited. Future progress will likely require rigorous clinical outcomes and rational combination of multimodal treatment strategies.

Duchenne muscular dystrophy (DMD) is a rare X-linked genetic disorder caused by mutations in the dystrophin gene, leading to an almost complete absence of dystrophin, which is essential for muscle cell structure and function. This resulting muscle deterioration and fibrosis, eventually causes respiratory failure and cardiomyopathy. While there is currently no cure, existing therapies aim to prolong survival and alleviate symptoms. This paper reviews current and emerging therapies for DMD, focusing on their safety and efficacy. Although corticosteroids remain the standard treatment, newly approved drugs such as exon-skipping therapies, vamorolone, delandistrogene moxeparvovec, and givinostat provide new treatment options. Additionally, future therapies, including gene therapy, stem cell treatments, and anti-fibrotic agents, show promise for clinical application. Advancements in DMD treatments have expanded patient options. While gene therapy offers potential for correcting the genetic defect and alleviating symptoms, corticosteroids remain the most cost-effective and well-researched treatment. This is partly due to the lack of compelling long-term safety and efficacy data for gene therapies. The accelerated FDA review process has enabled faster approval of new medications; however many have provided minimal clinical benefit to patients. Despite these challenges, continued drug development and innovative research offer hope to patients.

Duchenne muscular dystrophy (DMD) is a X-linked genetic disorder characterized by progressive muscle degeneration and weakness, caused by mutations in the dystrophin gene. DMD has effects in early age with significantly shortened lifespan and deteriorated quality of life in the second decade creating an urgent need to develop better therapeutic options. Corticosteroid medication therapy is an integral tool for the management of DMD and several therapeutic options have been recently approved for use. A comprehensive literature search was completed to examine efficacy and safety profiles of the three corticosteroid medications available for use in DMD patients. The review presents information about the three agents through clinical trials, significant preclinical trials, and comparative studies. Managing DMD takes a multidisciplinary approach although long-term corticosteroid therapy remains a significant therapeutic tool. Based on the available published studies, unequivocal comparison between the benefits of the three medications cannot yet be made. When selecting a medication for a patient, the decision-making process will most likely rely on the minor differences in the adverse effect profiles. Whichever medication is utilized will surely be a part of a larger regimen that includes other novel therapeutic agents.

IntroductionDuchenne muscular dystrophy (DMD) is a progressive genetic disease leading to muscular weakness. DMD is caused by mutations of the dystrophin gene on the X chromosome that is responsible for production of dystrophin protein. Dystrophin contributes to structural support in muscle cells and mutations result in dystrophin protein deficiency which causes muscle damage and the associated clinical presentation. Exon skipping medications, including the exon 53 targeting viltolarsen, are the first agents with the ability to partially restore dystrophin protein.Areas coveredHerein, the authors profile viltolarsen for the DMD patients who are amenable to exon 53 skipping therapy and provide their expert perspectives on this subject.Expert opinionCurrent findings suggest that viltolarsen could play a role in the current and possible future treatment of DMD. Viltolarsen seems to be safe and restores dystrophin protein to around 6% of the normal level. Due to orphan drug status, after the completion of the phase 2 clinical trial, viltolarsen was granted accelerated approval in Japan and in the US. A phase 3 trial is currently in progress and needs to earn full approval. Although a multidisciplinary approach continues to be critical, the addition of exon skipping agents like viltolarsen may improve the quality of patients' lives. However, data on the long-term safety and efficacy of this medication are not yet available due to its recent accelerated approval.

Duchenne muscular dystrophy (DMD) is a progressive genetic disease characterized by muscular weakness with a global prevalence of 7.1 cases per 100,000 males. DMD is caused by mutations of the dyst...

In an effort to expedite the publication of articles related to the COVID-19 pandemic, AJHP is posting these manuscripts online as soon as possible after acceptance. Accepted manuscripts have been peer-reviewed and copyedited, but are posted online before technical formatting and author proofing. These manuscripts are not the final version of record and will be replaced with the final article (formatted per AJHP style and proofed by the authors) at a later time. The objective of this review is to detail the utility of statin medications in the prevention and treatment of intensive care unit (ICU) delirium. Delirium is a syndrome characterized by altered mental status, inattention, and disorganized thinking. It is particularly concerning in the ICU where specific risk factors are much more prevalent. Nonpharmacological therapy is the mainstay of treatment, aimed at increasing patient awareness; pharmacological therapies have also been explored with varying success. The utility of statin medications in this scenario has been investigated because of the numerous pleiotropic effects of these drugs. Although the benefits in terms of treating delirium are uncertain, statins may be good candidates for prevention. The peak anti-inflammatory effect of statins is delayed, so initiating a statin on admission will likely have little protective benefit, whereas continuation of a home regimen seems more likely to exert an effect. Statin medications are very commonly used, and, while their role in treating delirium is unclear, continuation of these medications from a home regimen is reasonable to decrease the odds of delirium in the intensive care population.

Introduction: Duchenne muscular dystrophy (DMD) is the result of X-chromosome-linked mutations to the dystrophin protein gene that prevent the normal development and repair of muscles leading to muscle deterioration. The condition affects nearly 1 in 3,500 males worldwide. Current therapeutics have not been sufficient in providing a cure or resulting in a significant extension in life expectancy, but many therapeutic options are currently under investigation. Areas covered: This article provides an overview of the current and emerging therapies for DMD giving particular focus to synthetic therapeutic options. The authors further provide their expert opinion. Expert opinion: Many discrepancies in primary outcomes of trials have led to questions of efficacy for medications, as well as difficulty in securing FDA approval. A standardization of primary outcome strategies, as well as better access to investigational medications, may alleviate some of the controversy and pressures that exist on medication approvals. Many trials have identified cohorts who responded more favorably to medications, despite a lack of significance in the overall intent-to-treat populations. This indicates that more medication screening and personalized treatment with patient-specific targeting might deliver more clinically significant results.

With the opioid epidemic reaching new heights in the USA, it has become critical to find suitable alternatives to opioids. Cannabis, an antinociceptive, is a strong contender to help patients reduce their opioid usage. A growing literature has been examining the complex effects cannabis has on pain relief and on opioid usage; whether it is a substitute for opioids or increases their use. This review explores the studies that compare cannabis-opioid interactions and presents some challenges of cannabis research and usage. The practical clinical pharmacology of cannabis as an analgesic, including the route of administration, safety and pharmacokinetics, are discussed to address the concerns, as well as possible solutions, of cannabis as a pain reliever.