Great Ormond Street Hospital Charity’s Post

Clene Nanomedicine, Inc.'s Vice President of Translational Medicine, Karen S. Ho, PhD, presented preliminary data demonstrating neuroprotective effects of #CNMAu8 in an in vitro model of Rett Syndrome, a rare pediatric neurological disorder, at the International Rett Syndrome Foundation 2024 Annual Meeting. In the study, CNM-Au8 rescued neuron health and improved mitochondrial respiration in Rett patient-derived astrocytes, demonstrating CNM-Au8’s potential to positively impact multiple neurological diseases. Learn more here: https://bit.ly/4bcOeKi

The lab of Assistant Professor Keren Lasker has been awarded the 2024 CDKL5 Program of Excellence Pilot Grant to investigate the role of the CDKL5 protein in neurodevelopmental disorders. This prestigious grant, offered by the LOULOU FOUNDATION in collaboration with the Orphan Disease Center | University of Pennsylvania, supports pioneering work to understand and treat CDKL5 Deficiency Disorder (CDD). The Lasker Lab’s funded project focuses on membraneless organelles (MLOs) to explore how CDKL5 mutations lead to severe developmental delays and early-onset epilepsy. This research could lead to new treatments for CDD and related neurological disorders. More: https://ow.ly/HlaN50Ss1JU

The Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) will be hosting a webinar on May 2, 2024 from 1-2 pm ET, to introduce their new two-stage application and review process for OTA-24-013, NeuroNEXT Clinical Trials Stage 1 Preliminary Application, and OTA-24-014, NeuroNEXT Clinical Trials Stage 2 Protocol Application. #NeuroNEXT provides robust, standardized, and accessible infrastructure, designed to facilitate development and conduct of #Phase2 #clinicaltrials or #biomarkerstudies focused on #neurologicaldisease in pediatric and adult populations. More about the event can be found here: https://lnkd.in/eHH7gRnH

Targeting TTX-sensitive and TTX-resistant sodium channels in DRG sensory neurons holds promise in pain therapeutics.  At our San Diego Cell Electrophysiology lab, we've perfected a robust Patch Clamp assay. We achieve a high success rate in recording TTX-R sodium channel currents in DRGs from various species, including NHPs. Our results unveil species-specific compound activities and validate several proprietary Nav1.7 and Nav1.8 inhibitors. Explore the potential of Cell Electrophysiology. Contact us at [email protected] to discuss your research needs.   #cellelectrophysiology #CNS #Pain #Paindrugdiscovery #CNS #drugdiscovery #neuroscience2023 #SfN2023 

It's #TEAMTESSTUESDAY and this week we interviewed Daniel Lowenstein, MD, a member on the TESS Scientific Advisory Board! 👏🤩 Q: How did you become involved with our Scientific Advisory Board (SAB)? A: The remarkable Kim Nye reached out and presented the chance to be a part of the SAB. The opportunity was too compelling to pass up, considering the dedication and zeal of the TESS team in advancing research and bettering the lives of those affected by SLC13A5 Epilepsy. Q: What is your current role outside of TESS Research Foundation? A: I am a Professor of Neurology at the University of California, San Francisco (UCSF), and have recently concluded an 8 year term as the Executive Vice Chancellor and Provost. I currently divide my time between helping to lead large-scale, team-science efforts exploring the genetic causes of epilepsy, seeing patients and teaching at San Francisco General Hospital, and doing quite a bit of mentoring and advising of trainees and junior faculty. Q: What inspires you to contribute your time and skills as an SAB member? A: It’s quite simple, really. I hold a firm belief that to achieve our ultimate goal of curing epilepsy, we must deepen our understanding of the human brain's complexities and the specific mechanisms by which SLC13A5 mutations cause seizures. Q: From your perspective, what are the most pressing challenges or opportunities facing our SLC13A5 community in terms of scientific research and development? A: The biggest challenge (and opportunity!) is to increase society’s prioritization of biomedical research by enlarging the infrastructure that will enable brilliant minds to engage in the research leading to cures. The creative potential of human beings is seemingly limitless, so our goal must be to encourage and provide training opportunities for talented young people to pursue research careers, and create the resources to enable scientific inquiry of the very highest quality. Q: What is one thing you have learned from volunteering with TESS Research Foundation? A: My involvement with the TESS team has been incredibly enlightening, showcasing the immense power of compassion, collaboration, and excellence. These values are central to TESS's mission and are crucial in driving us toward the ultimate goal of finding a cure for SLC13A5 Epilepsy. Thank YOU for being part of #TEAMTESS, Daniel! We are lucky to have you on our Scientific Advisory Board! 🙏💜 #TESSResearch #SLC13A5Epilepsy #RareDisease #Epilepsy #EpilepsyAwareness

Early neurodevelopmental assessments effectively identified infants with cerebral palsy and predicted its severity in extremely small or preterm infants and those with encephalopathy, according to a study adding real-world support to prior findings. Among 116 infants, the accuracy of diagnosis of cerebral palsy or high risk of cerebral palsy at a corrected age of 3 to 4 months was 85% (95% CI 77-91) overall and 81% (95% CI 73-88) for predicting moderate-to-severe cases when compared with results at 24 to 36 months' corrected age, reported Abdul Razak, MD, of Monash University in Melbourne, Australia, and colleagues. However, when it came to predicting cognitive impairment, the accuracy of early neurodevelopmental assessments was "notably limited," ranging from 44% to 60%, the authors wrote in JAMA Network Openopens in a new tab or window. The findings were also presented at the Pediatric Academic Societies meetingopens in a new tab or window in Toronto.   https://lnkd.in/eBG_hu92 

📈 Trending Article in PNAS Nexus One of the most-viewed PNAS Nexus articles in the last week is “p17/C18-ceramide–mediated mitophagy is an endogenous neuroprotective response in preclinical and clinical brain injury.” Explore the findings here: https://ow.ly/CJ9O50QE3C4 To learn more about submitting your own research, visit PNAS Nexus at https://ow.ly/T3oH50QE3C3

Gel4Med is thrilled to announce a three-year $1.75M collaborative grant awarded by the Congressionally Directed Medical Research Programs (CDMRP)/Department of Defense (DoD) to evaluate new therapeutic approaches with the potential to revolutionize the treatment landscape in spinal cord injury (SCI). The research will focus on a new method of cell therapy, using Gel4Med’s proprietary Smart Materials Platform to deliver Schwann cells, a type of nerve cell, to the injury site on spinal cords. The research will be led by principal investigator, Damien Pearse, PhD, a nationally recognized scholar in the field of spinal cord injury, at the University of Miami/Miami Project to Cure Paralysis and co-investigator, Manav Mehta, PhD (Gel4Med). https://lnkd.in/ey4tX6Gv #Gel4Med #Smartbiomaterials #researchandinnovation #collaboration