Review
Clinical Neurology
Shujun Chen, Tianyu Liang, Tao Xue, Shouru Xue, Qun Xue
Summary: Pridopidine was found to improve motor scores in HD patients, with no significant adverse effects observed in both short-term and long-term use. However, when the dosage exceeded 90 mg/day, improvements in motor scores were seen but with increased risk of adverse events, such as nasopharyngitis and insomnia. Further randomized controlled trials are needed to evaluate the efficacy and safety of pridopidine in Huntington's disease.
FRONTIERS IN NEUROLOGY
(2021)
Article
Clinical Neurology
Douglas R. Langbehn, Elisabeth M. Fine, Andreas Meier, Steven Hersch
Summary: This study evaluates the normalized prognostic index (PIN) score as a tool for selecting participants for a perimanifest trial and explores the anticipated inclusion rates based on PIN thresholds. The results show that the composition of a perimanifest clinical trial population can be defined using preselected PIN thresholds, facilitating the assessment of potential disease-modifying therapies.
MOVEMENT DISORDERS
(2022)
Article
Clinical Neurology
Amrita Mohan, Zhaonan Sun, Soumya Ghosh, Ying Li, Swati Sathe, Jianying Hu, Cristina Sampaio
Summary: A study developed and validated a model of Huntington's disease progression using machine-learning methods, identifying nine disease states and revealing transition probabilities ranging from 5% to 27%. The findings can improve trial design and participant selection.
MOVEMENT DISORDERS
(2022)
Article
Cardiac & Cardiovascular Systems
Christoffer Rasmus Vissing, Anna Axelsson Raja, Sharlene M. Day, Mark W. Russell, Kenneth Zahka, Harry M. Lever, Alexandre C. Pereira, Steven D. Colan, Renee Margossian, Anne M. Murphy, Charles Canter, Richard G. Bach, Matthew T. Wheeler, Joseph W. Rossano, Anjali T. Owens, Lee Benson, Luisa Mestroni, Matthew R. G. Taylor, Amit R. Patel, Ivan Wilmot, Philip Thrush, Jonathan H. Soslow, Jason R. Becker, Christine E. Seidman, Neal K. Lakdawala, Allison L. Cirino, John J. V. Mcmurray, Calum A. Macrae, Scott D. Solomon, Henning Bundgaard, E. John Orav, Carolyn Y. Ho
Summary: This study explored the potential of valsartan to modify disease development in subclinical HCM and characterized the short-term phenotypic progression. The results showed that valsartan did not have a significant effect on cardiac remodeling and the progression of subclinical HCM was minimal. Nearly one-fifth of the patients developed clinically overt HCM, and the transition to disease was associated with baseline interventricular septum thickness and left atrial volume index. These findings emphasize the importance of longitudinal follow-up of sarcomere variant carriers and the need to improve understanding of factors that drive disease penetrance and progression.
Article
Oncology
Jelle E. Bousema, Marcel G. W. Dijkgraaf, Erik H. F. M. van der Heijden, Ad F. T. M. Verhagen, Jouke T. Annema, Frank J. C. van den Broek
Summary: The study suggests that confirmatory mediastinoscopy can be omitted in patients with resectable NSCLC who have no evidence of mediastinal metastases on systematic endosonography.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Medicine, General & Internal
Shivan J. Mehta, Colleen Mallozzi, Pamela A. Shaw, Catherine Reitz, Caitlin McDonald, Matthew Vandertuyn, Mohan Balachandran, Michael Kopinsky, Christianne Sevinc, Aaron Johnson, Robin Ward, Sae-Hwan Park, Christopher K. Snider, Roy Rosin, David A. Asch
Summary: This study aimed to evaluate the effectiveness of using text messaging to increase COVID-19 vaccine uptake. The results showed that there was no significant increase in vaccination rates among patients who received text messages compared to those who received telephone calls or behaviorally informed messages.
Article
Medicine, General & Internal
Stephen J. Nicholls, Steven E. Nissen, Cynthia Fleming, Shweta Urva, Jeffrey Suico, Paul H. Berg, Helle Linnebjerg, Giacomo Ruotolo, P. Kellie Turner, Laura F. Michael
Summary: The study determined the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of Muvalaplin. Results showed that Muvalaplin, as a selective small molecule inhibitor of Lp(a) formation, decreased Lp(a) levels by up to 65% following daily administration for 14 days. Longer and larger trials are needed to evaluate the impact of Muvalaplin on Lp(a) levels and cardiovascular outcomes.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2023)
Editorial Material
Clinical Neurology
Tanya N. Turan, Marios-Nikos Psychogios
Summary: The CASSISS trial, published in JAMA, adds to the growing body of evidence that percutaneous angioplasty and stenting does not provide a benefit over medical therapy for preventing stroke due to intracranial atherosclerotic stenosis. Current guidelines recommend against routine use of this intervention for stroke prevention in these patients. The trial may offer valuable insights for future trial design.
Article
Psychiatry
Nick Medford, Natasha Sigala
Summary: We present a case of a patient with Huntington's disease who developed clinical lycanthropy, a delusion of being a werewolf, following a history of anxiety and obsessive preoccupations. The patient did not respond to typical treatment options but showed remarkable improvement with Clozapine. This is the first reported case of clinical lycanthropy in a patient with Huntington's disease, highlighting the potential role of cultural and personal factors in the manifestation of this delusion.
FRONTIERS IN PSYCHIATRY
(2023)
Article
Medicine, General & Internal
Neha J. Pagidipati, Adam J. Nelson, Lisa A. Kaltenbach, Monica Leyva, Darren K. McGuire, Rodica Pop-Busui, Matthew A. Cavender, Vanita R. Aroda, Melissa L. Magwire, Caroline R. Richardson, Ildiko Lingvay, Julienne K. Kirk, Hussein R. Al-Khalidi, Laura Webb, Tanya Gaynor, Jonathan Pak, Cagri Senyucel, Renato D. Lopes, Jennifer B. Green, Christopher B. Granger
Summary: This study aimed to assess the effect of a coordinated, multifaceted intervention on the treatment of patients with type 2 diabetes and atherosclerotic cardiovascular disease. The results showed that the intervention significantly increased the proportion of patients prescribed medication and they were more likely to receive each treatment. Therefore, a multifaceted intervention can effectively improve the treatment level of patients with type 2 diabetes and atherosclerotic cardiovascular disease.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2023)
Article
Clinical Neurology
Jamie L. Hamilton, James A. Mills, Glenn T. Stebbins, Jeffrey D. Long, Rebecca L. M. Fuller, Swati Sathe, Matt Roche, Cristina Sampaio
Summary: This study aims to estimate the minimal clinically important difference (MCID) for clinically relevant outcome measures in individuals with Stages 2 or 3 Huntington's disease. By analyzing data from a large global longitudinal study, the study found that MCID estimates increased with disease progression and longer time frames. These estimates can improve the clinical interpretation of study outcomes and support clinical decision-making and trial methodology.
MOVEMENT DISORDERS
(2023)
Review
Immunology
Qingqing Jia, Shihua Li, Xiao-Jiang Li, Peng Yin
Summary: Huntington's disease is a progressive neurodegenerative disorder characterized by loss of neurons in the striatum, leading to motor and cognitive impairments. The cause of the disease is the expansion of a glutamine repeat in the huntingtin protein, but the mechanism underlying neuronal death is still unclear. Inflammation is one of the contributing factors in the pathogenesis of Huntington's disease. This review summarizes the evidence on immune and inflammatory changes in Huntington's disease, providing insights for the development of novel strategies for its treatment.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Clinical Neurology
Rachid Riad, Marine Lunven, Hadrien Titeux, Xuan-Nga Cao, Jennifer Hamet Bagnou, Laurie Lemoine, Justine Montillot, Agnes Sliwinski, Katia Youssov, Laurent Cleret de Langavant, Emmanuel Dupoux, Anne-Catherine Bachoud-Levi
Summary: Using machine learning and speech features, we were able to predict the clinical performance of Huntington's Disease patients, which could be an efficient method for remote evaluation of individual condition in the future.
JOURNAL OF NEUROLOGY
(2022)
Article
Biochemistry & Molecular Biology
Dhiraj Kumar, Gulam Mustafa Hasan, Asimul Islam, Md. Imtaiyaz Hassan
Summary: Huntington's disease is a common autosomal dominant disease that affects a large number of patients. Despite early genetic diagnostics, effective disease-modifying treatments are still lacking. Ongoing clinical trials are exploring potential drugs to alleviate symptoms by targeting the root cause at the molecular level. Although a major phase III trial was unexpectedly discontinued, there is still optimism about the potential of this technique.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
(2023)
Article
Cardiac & Cardiovascular Systems
Sherrie Khadanga, Patrick D. Savage, Anton Pecha, Jason Rengo, Philip A. Ades
Summary: In this randomized clinical trial, a combination of HIIT and intensive lower extremity RT showed enhanced exercise training response for women in cardiac rehabilitation compared to standard exercise training, with significantly greater improvements in both peak Vo(2) and leg strength in the HIIT group.
Article
Clinical Neurology
Mathias Granqvist, Joachim Burman, Martin Gunnarsson, Jan Lycke, Petra Nilsson, Tomas Olsson, Peter Sundstrom, Anders Svenningsson, Magnus Vrethem, Thomas Frisell, Fredrik Piehl
MULTIPLE SCLEROSIS JOURNAL
(2020)
Article
Clinical Neurology
Gustavo Luna, Peter Alping, Joachim Burman, Katharina Fink, Anna Fogdell-Hahn, Martin Gunnarsson, Jan Hillert, Annette Langer-Gould, Jan Lycke, Petra Nilsson, Jonatan Salzer, Anders Svenningsson, Magnus Vrethem, Tomas Olsson, Fredrik Piehl, Thomas Frisell
Article
Endocrinology & Metabolism
Henrik Carlsson, Sandy Abujrais, Stephanie Herman, Payam Emami Khoonsari, Torbjorn Akerfeldt, Anders Svenningsson, Joachim Burman, Kim Kultima
Article
Clinical Neurology
Joakim Bergman, Joachim Burman, Tommy Bergenheim, Anders Svenningsson
Summary: The study evaluated the effect of intrathecally delivered rituximab on PMS and found that it was well tolerated but did not halt disease progression.
JOURNAL OF NEUROLOGY
(2021)
Article
Biochemistry & Molecular Biology
Henrik Carlsson, Niclas Rollborn, Stephanie Herman, Eva Freyhult, Anders Svenningsson, Joachim Burman, Kim Kultima
Summary: This study analyzed the metabolomes of healthy individuals' CSF and identified ten metabolites significantly associated with ageing, with eight increasing with age and two decreasing with age. These findings contribute to the field of ageing metabolomics and provide motivation for further in-depth research.
Editorial Material
Clinical Neurology
Victoria M. Leavitt, Ruth Dobson, Anders Svennigsson
Article
Clinical Neurology
Tim Spelman, Melinda Magyari, Fredrik Piehl, Anders Svenningsson, Peter Vestergaard Rasmussen, Matthias Kant, Finn Sellebjerg, Hanna Joensen, Jan Hillert, Jan Lycke
Summary: This study investigated the association between national differences in treatment strategies for RRMS and disability outcomes, finding that the Swedish treatment strategy was associated with reduced rates of disability worsening compared to the Danish strategy.
Article
Pharmacology & Pharmacy
Erik Rein-Hedin, Folke Sjoberg, Susanna Waters, Clas Sonesson, Nicholas Waters, Fredrik Huss, Joakim Tedroff
Summary: The cortical enhancer Pirepemat showed good tolerability and pharmacokinetic properties in a first-in-human study, supporting its further clinical development for the prevention of falls in Parkinson disease patients.
CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT
(2021)
Article
Clinical Neurology
Anders Svenningsson, Thomas Frisell, Joachim Burman, Jonatan Salzer, Katharina Fink, Susanna Hallberg, Joakim Hambraeus, Markus Axelsson, Faiez Al Nimer, Peter Sundstrom, Martin Gunnarsson, Rune Johansson, Johan Mellergard, Igal Rosenstein, Ahmad Ayad, Irina Sjoblom, Anette Risedal, Pierre de Flon, Eric Gilland, Jonas Lindeberg, Fadi Shawket, Fredrik Piehl, Jan Lycke
Summary: Rifund-MS study provides evidence that rituximab is superior to dimethyl fumarate in preventing relapses in patients with early relapsing-remitting multiple sclerosis. Adverse events such as infusion reactions and gastrointestinal reactions were observed, but there were no safety concerns.
Editorial Material
Clinical Neurology
Jan Lycke, Anders Svenningsson
MULTIPLE SCLEROSIS JOURNAL
(2022)
Article
Clinical Neurology
Elisa Longinetti, Hannah Bower, Kyla A. McKay, Simon Englund, Joachim Burman, Katharina Fink, Anna Fogdell-Hahn, Martin Gunnarsson, Jan Hillert, Annette Langer-Gould, Jan Lycke, Petra Nilsson, Jonatan Salzer, Anders Svenningsson, Johan Mellergard, Tomas Olsson, Fredrik Piehl, Thomas Frisell
Summary: This study investigated the risks and associations with disease-modifying therapies in multiple sclerosis patients during the COVID-19 pandemic. The results showed that multiple sclerosis patients had increased risks for COVID-19 and other severe outcomes compared to population controls, but these risk increases were not significantly different from previous years. The risk conveyed by disease-modifying therapies was smaller than previously assumed, likely due to better control for confounding factors.
ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY
(2022)
Article
Medicine, General & Internal
Ida Petersson, Bjorn M. Hansen, Anders Svenningsson, Annika Lundstrom
Summary: Neuroradiological findings in COVID-19 patients can resemble cerebral small vessel disease (CSVD). We present a case of a severe COVID-19 patient who showed white matter abnormalities and restricted diffusion on MRI. The patient experienced significant clinical recovery but MRI showed progression of the white matter abnormalities.
Article
Clinical Neurology
H. Tedeholm, F. Piehl, J. Lycke, J. Link, L. Stawiarz, J. Burman, P. de Flon, K. Fink, M. Gunnarsson, J. Mellergard, P. Nilsson, P. Sundstrom, A. Svenningsson, H. Johansson, O. Andersen
Summary: This study examined the impact of disease-modifying therapies (DMTs) on the risk of transition to secondary progressive MS (SPMS) using nationwide population-based data in Sweden. The results showed that the introduction of DMTs significantly decreased the incidence of SPMS conversion. The age at onset played a role in determining the risk of SPMS, and this pattern was observed in both treated and untreated subgroups.
MULTIPLE SCLEROSIS AND RELATED DISORDERS
(2022)
Article
Pharmacology & Pharmacy
Folke Sjoberg, Susanna Waters, Boel Lofberg, Clas Sonesson, Nicholas Waters, Joakim Tedroff
Summary: The study evaluated the safety, tolerability, and pharmacokinetics of mesdopetam in healthy male subjects, demonstrating good safety and tolerability, rapid absorption, and dose-linear pharmacokinetics. These findings support the potential use of mesdopetam in patients with Parkinson's disease.
PHARMACOLOGY RESEARCH & PERSPECTIVES
(2021)
Article
Clinical Neurology
L. Novakova, M. Axelsson, C. Malmestrom, H. Zetterberg, K. Blennow, A. Svenningsson, J. Lycke
MULTIPLE SCLEROSIS AND RELATED DISORDERS
(2020)