An overview of recent US-approved gene therapies for Duchenne muscular dystrophy and their respective clinical development programs
AbstractThere is no current cure for Duchenne muscular dystrophy (DMD), a rare genetic disease in young male patients, and the aim of treatment is to delay disease progression. Recent advancements in gene therapy provide potential improvement in targeting the underlying cause of DMD. Since 2016, the US FDA has approved four new antisense oligonucleotides for the treatment of DMD that are designed to achieve the stated objective and attenuate disease symptoms. They were registered via the accelerated pathway due to the life-threatening nature of the disease, unmet medical need, and possession of a unique mechanism of action...
Source: Drugs and Therapy Perspectives - March 21, 2023 Category: Drugs & Pharmacology Source Type: research
Ventricular arrhythmias, antiarrhythmic therapy and thyroidal illness in advanced heart failure: a case report and review of the literature
We describe the clinical course of antiarrhythmic therapy and the resultant complication of clinically significant thyroid dysfunction. We also summarize our review of the literature regarding the treatment of ventricular arrhythmias in advanced heart failure and the management of antiarrhythmic therapy-induced thyroid dysfunction. (Source: Drugs and Therapy Perspectives)
Source: Drugs and Therapy Perspectives - March 2, 2023 Category: Drugs & Pharmacology Source Type: research
Prescribe proton pump inhibitors with care and be aware of the signs of nephrotoxicity
This article discusses the incidences of adverse kidney events among PPI users, mechanisms that may underlie potential nephrotoxicity, and signs and risk factors clinicians should be aware of to ensure timely diagnosis and treatment. Care should be taken that PPIs are prescribed only with a clear indication for use and at an appropriate dose and duration; deprescription could be considered in some patients. (Source: Drugs and Therapy Perspectives)
Source: Drugs and Therapy Perspectives - February 24, 2023 Category: Drugs & Pharmacology Source Type: research
Nivolumab/relatlimab in advanced melanoma: a profile of its use
AbstractNivolumab and relatlimab-rmbw (Opdualag™; hereafter referred to as nivolumab/relatlimab) is a fixed dose combination of monoclonal antibodies for infusion, which expands the number of available therapies against advanced melanoma in patients aged ≥ 12 years. Dual checkpoint inhibition of programmed cell death protein-1 (PD-1) by nivolumab and lymphocyte-activation gene 3 (LAG-3) by relatlimab may stimulate an immune response against tumours. Notably, relatlimab is the first approved treatment targeting LAG-3. Nivolumab/relatlimab significa ntly decreased the risk of disease progression or death in comparison wi...
Source: Drugs and Therapy Perspectives - February 21, 2023 Category: Drugs & Pharmacology Source Type: research
Clozapine is the approved option in treatment-resistant schizophrenia and requires careful management
AbstractClozapine is the only agent approved for treatment-resistant schizophrenia, but is underprescribed. Its adverse drug event (ADE) profile and patient monitoring requirements can discourage its use, but the benefits of clozapine generally outweigh its risks, as most ADEs are manageable. Careful patient assessment, gradual titration, minimum effective dosages, therapeutic drug monitoring and checks of neutrophils, cardiac enzymes and ADE symptoms are recommended. Neutropenia is common but does not necessarily warrant permanent clozapine cessation. (Source: Drugs and Therapy Perspectives)
Source: Drugs and Therapy Perspectives - February 17, 2023 Category: Drugs & Pharmacology Source Type: research
Manage methamphetamine-associated cardiomyopathy by promoting abstinence in addition to the use of pharmacotherapies
AbstractMethamphetamine-associated cardiomyopathy (MAC) is on the rise. The treatment guidelines for heart failure do not fully take into consideration the comorbidities that are common in patients with MAC, and the safety and efficacy of recommended pharmacotherapies is unproven in these patients. Instead, achieving and maintaining abstinence from methamphetamine is the most important factor for recovery from MAC, and this requires a multidisciplinary approach which involves pharmaceutical interventions and psychosocial support. (Source: Drugs and Therapy Perspectives)
Source: Drugs and Therapy Perspectives - February 15, 2023 Category: Drugs & Pharmacology Source Type: research
The role of community pharmacists in optimising patient self-management of constipation: an inter-disciplinary consensus view
The objective of this article is to provide up-to-date clinical practice guidance for healthcare professionals, particularly community pharmacists, to assist in the day-to-day management of patients who present with constipation. Here, we discuss typical clinical factors (e.g. symptom duration, warning symptoms and treatment response) that h elp to distinguish whether constipation can be self-managed or if further medical assessment is required. We have also developed screening questions and a patient management algorithm to help pharmacists and pharmacy technicians make appropriate decisions when a patient presents with s...
Source: Drugs and Therapy Perspectives - January 31, 2023 Category: Drugs & Pharmacology Source Type: research
Ganaxolone in seizures associated with cyclin-dependent kinase-like 5 deficiency disorder: a profile of its use in the USA
AbstractGanaxolone (ZTALMY®), a synthetic neuroactive steroid that acts as a positive allosteric modulator of the synaptic and extrasynaptic gamma-aminobutyric acid (GABA)A receptor, is an effective and well tolerated, orally-administered adjunct to existing antiseizure treatments in patients 2 years of age or older with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD)-associated seizures. CDD (a rare X-linked genetic disorder) is a severe developmental epileptic encephalopathy with onset in early infancy. In the randomized, double-blind, phase 3 Marigold trial, adjunctive ganaxolone significantly reduced ...
Source: Drugs and Therapy Perspectives - January 30, 2023 Category: Drugs & Pharmacology Source Type: research
Growth-related effects of antiseizure medications are of concern in children with epilepsy
AbstractEpilepsy is a common chronic neurological condition in children that is treated with antiseizure medications as first-line (and subsequent-line) therapy. Sometimes the underlying cause of epilepsy can be associated with aberrant growth. Antiseizure medications can also increase or decrease the appetite, weight and growth in children with epilepsy. If an antiseizure medication is suspected of impacting appetite and growth, options include dose reduction, switching to another antiseizure medication and adding supportive options. However, these should be considered after weighing the benefits versus the risks of inter...
Source: Drugs and Therapy Perspectives - January 26, 2023 Category: Drugs & Pharmacology Source Type: research
First reports of adverse drug reactions
(Source: Drugs and Therapy Perspectives)
Source: Drugs and Therapy Perspectives - January 26, 2023 Category: Drugs & Pharmacology Source Type: research
Take a fresh look at diet and atopic dermatitis
AbstractThe links between atopic dermatitis (AD) and food allergies are complex, with impaired skin barrier function suspected to play a role in both conditions. Additionally, AD may be the first stage towards other atopic or allergic conditions, such as food allergies and asthma. Thus, early intervention in AD has the potential to interrupt this progression. Contrary to common understanding, large trials support the consumption of allergenic food (e.g. peanuts, dairy products and wheat) in infants to reduce the incidence of food allergies. Supplementing diets with fatty acids and other supplements may decrease the risk of...
Source: Drugs and Therapy Perspectives - January 24, 2023 Category: Drugs & Pharmacology Source Type: research
Avacopan in granulomatosis with polyangiitis and microscopic polyangiitis: a profile of its use
In this study, overall infections (a major driver of early mortality in this patient population), serious infections and serious opportunistic infections were less frequent with avacopan than tapered prednisone. (Source: Drugs and Therapy Perspectives)
Source: Drugs and Therapy Perspectives - December 20, 2022 Category: Drugs & Pharmacology Source Type: research
Methodically manage pain in older patients with Parkinson ’s disease
AbstractPain is a common, but often overlooked, non-motor symptom of Parkinson ’s disease (PD), with nociceptive pain being most common. Structured questionnaires may help clarify the origin, nature and impact of pain in patients with PD. Subject to comorbidities, polypharmacy and tolerability in older patients, recommended management includes optimisation of dopaminergic dr ugs, the use of safinamide or administration of analgesia, starting with paracetamol. Short-term oxycodone/naloxone, gabapentin or pregabalin may be needed in some patients. (Source: Drugs and Therapy Perspectives)
Source: Drugs and Therapy Perspectives - December 13, 2022 Category: Drugs & Pharmacology Source Type: research
Antidepressants with anti-inflammatory properties may be useful in long COVID depression
AbstractLong COVID, which is characterised by the presence of persistent symptoms following a COVID infection, may also cause long COVID depression (LCD). Although the risk factors for LCD are not directly characterised, prior mental health visits were associated with an increased risk for long COVID, whereas antidepressant use was not. Current evidence suggests that pro-inflammatory factors in the brain are linked to LCD, thus anti-inflammatory agents may be useful in mitigating LCD. Limited evidence suggests that selective serotonin reuptake inhibitors may also be effective in the treatment of LCD. (Source: Drugs and Therapy Perspectives)
Source: Drugs and Therapy Perspectives - December 12, 2022 Category: Drugs & Pharmacology Source Type: research
Treatment satisfaction and limitations in haemophilia A, with a focus on factor VIII product storage conditions: patients ’ perspectives and challenges
ConclusionThis survey highlights patient perspectives regarding treatment satisfaction and limitations in haemophilia A, with a focus on product storage conditions. Perceived product storage requirements limit the daily activities of many patients (especially travel). Ongoing patient education is necessary to improve treatment satisfaction, compliance, treatment outcomes and overall health. (Source: Drugs and Therapy Perspectives)
Source: Drugs and Therapy Perspectives - December 8, 2022 Category: Drugs & Pharmacology Source Type: research
