We speak to Francesco Danilo Tiziano about newborn screening for spinal muscular atrophy, including his experience in Italy and the impact of screening on patient outcomes.
Mariacristina Scoto talks about real-world studies on disease-modifying treatments for spinal muscular atrophy, and how some of the data presented at EPNS 2022 could impact clinical practice.
Laurent Servais talks about the challenges in treatment decision making for spinal muscular atrophy and how they could be addressed, drawing on early-phase anti-myostatin antibody research as a potential treatment solution if shown to be beneficial in phase 3 trials.
Author: John Wilding The clinical challenge Glucocorticoids, which include long-acting corticosteroid preparations such as prednisolone and dexamethasone, are frequently used to treat inflammatory conditions such as asthma and exacerbations of chronic obstructive pulmonary disease, and other conditions including many autoimmune diseases, and also to reduce cerebral edema in association with brain tumors. More recently, the […]
Peter Nash discusses the latest evidence on the association between JAK inhibitors and the risk for VTE, MACE, and malignancy medwireNews: Janus kinase (JAK) inhibitors are the newest class of drugs for the treatment of rheumatoid arthritis (RA), with three JAK inhibitors now approved by the US FDA and four by the EMA (figure 1). […]
Author: Shreeya Nanda The treatment landscape of advanced urothelial carcinoma has changed considerably in the past few years, with immunotherapy becoming a key component. In this article, we provide an overview of the published trials of PD-1 pathway and CTLA-4 inhibitors – used as monotherapy, in combination with chemotherapy, or as maintenance – in the […]
The FDA has recently highlighted the elevated risk for suicide in people with diabetes, resulting in the creation of the REducing SuiCide rates amongst individUals with diabEtes (RESCUE) Collaborative Community to examine issues relating to the identification and management of increased suicide risk in people with diabetes. In this brief overview, we look at the […]
Author: Hannah Kitt The PD-L1 inhibitor avelumab (formerly MSB0010718C) is being evaluated in the JAVELIN clinical trials as a single agent or in combination with an assortment of anticancer agents for the treatment of various malignancies. Here is a quick guide to the nine JAVELIN trials investigating avelumab in patients with non-small-cell lung cancer (NSCLC), […]
Author: Claire Barnard Challenges, guidelines, and research priorities Psoriatic arthritis (PsA), a heterogeneous disease involving multiple domains, can be challenging to manage, particularly in resource-limited settings. With the majority of published research studies in PsA coming from resource-rich countries, guidelines from organizations including the European League Against Rheumatism (EULAR), the American College of Rheumatology (ACR), […]
Author: Shreeya Nanda This article provides a top-line overview of the CheckMate clinical trials of the PD-1 inhibitor nivolumab – either given alone or alongside the CTLA-4 inhibitor ipilimumab – in patients with renal cell carcinoma (RCC). There are nine trials at the time of writing, all of which are sponsored by the drug manufacturer […]