CMTA-STAR Alliance Partner NMD Pharma A/S has announced the publication of foundational findings in the Annals of Clinical and Translational Neurology. The study highlights neuromuscular junction (NMJ) dysfunction as a characteristic in Charcot-Marie-Tooth disease (CMT) types 1 and 2, identifying its impact on muscle function and disease severity. This research laid the groundwork for NMD Pharma’s SYNAPSE-CMT Phase II clinical trial, which is now actively recruiting participants. Learn more: https://lnkd.in/eeKh74kw #CMTResearch #CMTASTAR #CharcotMarieTooth #CMTDisease #LetsBeatCMT #CMTAFamily
Charcot-Marie-Tooth Association’s Post
More Relevant Posts
-
📢 Thrilled to share one of our recent contributions to the scientific community! 🧠 Our review article, “Central Post-Stroke Pain: Advances in Clinical and Preclinical Research”, was published in Stroke & Vascular Neurology. This comprehensive review explores the clinical characteristics, evaluation methods, and therapeutic strategies for central post-stroke pain (CPSP). It bridges the gap between clinical findings and preclinical models, offering insights into the condition’s mechanisms and future directions for treatment development. It’s a privilege to collaborate with such talented researchers, and I hope this work can guide further advancements in understanding and managing CPSP.
To view or add a comment, sign in
-
A paper published in Nature Reviews Neurology, discusses suspected non-AD pathophysiology (SNAP), which is common in individuals with MCI and normal cognition (NC). People with NC-SNAP have a low risk of cognitive decline over 7 years, and longer follow-up studies are needed to demonstrate whether they will eventually show faster cognitive decline than those with NC and normal biomarkers. People with MCI-SNAP have an increased risk of developing clinical AD dementia at follow-up (annual conversion rate 7% compared with 1% for MCI with normal biomarkers). This finding suggests that SNAP might partly reflect a lack of sensitivity of biomarkers to detect early amyloid pathology, although misdiagnosis at follow-up is another possibility. The frequency of AD genetic risk factors in A− T+ individuals with SNAP is similar to that in control individuals and lower than in those with AD. This finding is mainly driven by a lower frequency of APOE ε4 in SNAP than in AD. CSF proteomics have shown that people with SNAP have increased CSF levels of Aβ38 and Aβ40 compared with patients with AD and control individuals, possibly reflecting the effects of tau pathology without marked amyloid aggregation. A distinguishing feature in people with MCI-SNAP is a decrease in CSF levels of proteins that are highly expressed in the choroid plexus, suggesting a role for this structure in MCI-SNAP. Neuropathological studies have indicated a range of possible neuropathologies associated with SNAP, including TDP43-related pathologies (LATE and FTLD-TDP), tau pathology (PART, AGD and PSP), LBD and cerebrovascular pathology. In older individuals, SNAP probably represents a combination of neuropathological lesions. Nature Portfolio Springer Nature Group #neuroscience #neurology #neurologicaldisorders #neurodegenerativedisorders #alzheimersdisease #alzheimer #cognitivedecline #dementia #biomarkers #precisionmedicine #diagnosis #earlydiagnosis #earlydetection #prediction #SNAP #PART #FTD #ALS #Parkinsonsdisease #AGD #PSP #LATE #neuropathology #brain #brainresearch #clinicalresearch #MCI #cognition #amyloid #pathophysiology #riskfactors #classification #differentialdiagnosis
I’m thrilled to share our recent review and meta-analysis on the clinical importance of Suspected Non-Alzheimer disease Pathophysiology (SNAP) that is now published in Nature Reviews Neurology. SNAP is a heterogeneous biomarker-based concept that describes individuals with normal amyloid and abnormal tau and/or neurodegeneration biomarker status. In this review, we describe the origins of the SNAP construct, along with its prevalence, diagnostic and prognostic implications, and underlying neuropathology. You can read the full paper here: https://rdcu.be/dHrrE Special thanks to Aurore Delvenne Clifford Jack Dietmar Thal Pieter Jelle Visser
To view or add a comment, sign in
-
-
Discover the Future of CNS Therapeutics: The Promise of KCC2 Activation Join us on November 13 as we explore a breakthrough in neuroscience, focusing on a critical target in the central nervous system (CNS) – KCC2. This unique transporter, found exclusively in the brain, plays a fundamental role in controlling neuron excitability. Activating KCC2 holds transformative potential for treating a wide spectrum of neurological conditions by addressing the underlying mechanisms that drive disorders such as epilepsy, psychosis, and mood disorders. Ovid Therapeutics is at the forefront of advancing this field. We are proud to have developed multiple KCC2 activators and are on track to submit our first IND for OV350, a promising candidate that could open doors to life-changing therapies for those affected by CNS diseases. I’ll be joined by esteemed experts, Professors Stephen Moss, Jeffrey Noebels, and Karl Kieburtz, along with key leaders from Ovid, to discuss the groundbreaking science behind KCC2 activation. Together, we’ll delve into how this new approach could reshape treatment paradigms for brain disorders. Don’t miss this chance to engage with a new frontier in small molecule neurotherapeutics and witness the potential impact of KCC2 on the future of brain health. Save the Date: November 13 Be part of the conversation that could redefine the future of CNS treatments. Register to join in person: https://lnkd.in/dvZvmvCc Register to join the virtual webcast: https://lnkd.in/dWJfKxkV
We’re honored to welcome three recognized thought leaders in neurology and neuropharmacology to present at Ovid’s KCC2 Download Day next week. Stephen Moss, Jeff Noebels and Karl Kieburtz will discuss the biological functions, underlying mechanisms and therapeutic promise of directly activating KCC2. KCC2 is one of the most exciting novel pathways in neurology due to its role in modulating neuronal hyperexcitability and neuroinflammation, the root cause of many brain disorders. Join us on November 13 to explore the biological and therapeutic significance of KCC2 activation across a wide range of CNS conditions. Register to join in person: https://lnkd.in/eGzQqw9X Register to join the virtual webcast: https://lnkd.in/eCBq8gFC
To view or add a comment, sign in
-
-
The May 2024 S:VIN Journal is now available! Access it here: https://lnkd.in/gJ9wqUSy Stroke: Vascular and Interventional Neurology is a fully Open Access bi-monthly journal aimed primarily at reporting clinical, translational and basic sciences research on interventional, endovascular, medical and surgical management of stroke and vascular disease of the brain, spinal cord, and head and neck.
To view or add a comment, sign in
-
-
Zai Lab (NASDAQ: ZLAB, SEHK: 9688) has announced a study led by Professors Sushan Luo and Chongbo Zhao from Huashan Hospital, Fudan University, published in the Annals of Clinical and Translational Neurology. This research represents the first multi-center, real-world cohort study in China evaluating Efgartigimod for the treatment of generalized myasthenia gravis (gMG). Under an exclusive licensing agreement with argenx, Zai Lab is developing and commercializing Efgartigimod in Greater China to address significant unmet needs in autoimmune conditions. Key findings from the study include: - 97% of patients experienced clinically meaningful improvement within an average of 1.3±0.7 weeks. - Efgartigimod demonstrated efficacy across various antibody types and disease states, including MuSK antibody-positive, seronegative, acute exacerbation, and myasthenic crisis patients. - Approximately 70% of patients transitioned to a mild disease state, and 39% achieved minimal symptom expression after 12 weeks of follow-up. - The average daily dose of corticosteroids decreased from 28.3mg at baseline to 15.7mg over 12 weeks. #HealthcareInnovation #RareDiseases #ClinicalResearch #AutoimmuneDiseases #Neurology #Efgartigimod #QimingPortfolio #QimingHealthcare
To view or add a comment, sign in
-
-
Labs looking for biomarkers could really help us. We could not only treat disease but help with best targeted preventative health tips for everyone. It has the potential to give people more years with better quality of life. LessBarriers.com #PrevenativeHealth #Neurology #PersonalizedHealthcare #Biomarkers
Most viewed in the last 7 days from JAMA Neurology: This Viewpoint discusses using digital biomarkers for neurodegenerative disease. https://ja.ma/40n4q8d
To view or add a comment, sign in
-
-
Generalized myasthenia gravis is a chronic autoimmune disorder without a cure. A recent study by Zai Lab showed favorable results with Efgartigimod. Approximately 70% of patients transitioned to a mild disease state, with 39% achieving minimal symptom expression. These improvements are significant for patients and caregivers alike. #Qiming #QimingPortfolio #healthcare #ZaiLab #Clinical #TranslationalNerology #Efgartigimod #gMg #MyastheniaGravis #Argenx #medtech #biotech #Antibody
Zai Lab (NASDAQ: ZLAB, SEHK: 9688) has announced a study led by Professors Sushan Luo and Chongbo Zhao from Huashan Hospital, Fudan University, published in the Annals of Clinical and Translational Neurology. This research represents the first multi-center, real-world cohort study in China evaluating Efgartigimod for the treatment of generalized myasthenia gravis (gMG). Under an exclusive licensing agreement with argenx, Zai Lab is developing and commercializing Efgartigimod in Greater China to address significant unmet needs in autoimmune conditions. Key findings from the study include: - 97% of patients experienced clinically meaningful improvement within an average of 1.3±0.7 weeks. - Efgartigimod demonstrated efficacy across various antibody types and disease states, including MuSK antibody-positive, seronegative, acute exacerbation, and myasthenic crisis patients. - Approximately 70% of patients transitioned to a mild disease state, and 39% achieved minimal symptom expression after 12 weeks of follow-up. - The average daily dose of corticosteroids decreased from 28.3mg at baseline to 15.7mg over 12 weeks. #HealthcareInnovation #RareDiseases #ClinicalResearch #AutoimmuneDiseases #Neurology #Efgartigimod #QimingPortfolio #QimingHealthcare
To view or add a comment, sign in
-
-
Just Released! A publication featuring Malnati Brain Tumor Institute investigators Amy Heimberger, MD, PhD, and Adam Sonabend, MD, titled "Crossing the Blood-Brain Barrier: Emerging Therapeutic Strategies for Neurological Disease." The blood-brain barrier is a protective, semipermeable border between the central nervous system (CNS) and the circulatory system, which prevents substances in the blood from reaching the brain. This creates a challenge for delivering drugs to the brain for the treatment of neurological disorders. Dr. Heimberger and Dr. Sonabend collaborated with faculty from the University of Liverpool on this publication, focusing on different strategies to overcome the blood-brain barrier and deliver drugs effectively. The publication is featured in The Lancet Neurology and can be read by clicking the link below: https://lnkd.in/gXmd5z9m #bloodbrainbarrier #CSFDelivery #brainmetastasis #glioblastoma #epilepsy #multiplesclerosis #intracranialdelivery #nanotherapeutics
To view or add a comment, sign in
-
-
Most viewed in the last 7 days from JAMA Neurology: This Viewpoint discusses using digital biomarkers for neurodegenerative disease. https://ja.ma/3DYb25L
To view or add a comment, sign in
-
-
Thanks MassDevice for covering our recent paper in Frontiers in Neurology. Read the article by Sean Whooley: https://lnkd.in/eNXF4-GB
To view or add a comment, sign in