CurePSP’s Post

Did you know? There are an estimated 17-26k patients suffering Rett Syndrome in Australia, USA, Europe, and Japan. When combined with a limited number of Rett specialist clinicians, this highlights the critical need for innovative treatments. Our investigational drug, NTI164, has shown promising pre-clinical results in treating neuroinflammation, which is a critical factor in Rett Syndrome. In key studies, NTI164 reduced inflammatory markers by up to 80%. We are committed to advancing NTI164 through clinical trials to determine its safety and efficacy in patients with Rett Syndrome. We believe NTI164 has the potential to significantly improve the lives of those affected by this debilitating condition. For more information on NTI164, visit https://lnkd.in/dC35qwTi. #Neurotech #NTI ##NTI164 #neurologicalresearch #bettertogether #RettSyndrome #ASD #neurologicalresearch 

AlzeCure reports anti-inflammatory effects with NeuroRestore ACD856 with relevance to Alzheimer's leading to new patent application AlzeCure  has received preclinical data for NeuroRestore in inflammation, with relevance to Alzheimer's disease, and submitted a new patent application for NeuroRestore ACD856, which is now being prepared for phase II clinical studies. “We continue to strengthen the patent portfolio for our NeuroRestore program, and already have approved patents in all major markets such as the USA, Europe, Japan and China. The new results for our clinical drug candidate ACD856, which we are preparing for phase II against Alzheimer's, provide further evidence for a disease-modifying effect as well as general use against neurodegenerative diseases, where neuroinflammation is central. This further improves our commercial opportunities for this promising substance,” said Martin Jönsson, CEO. Read more here: https://lnkd.in/dTrg-DHy About the share issue: https://lnkd.in/d6PHyuRe #biostock #finwire #alzecure #alzheimers #alzheimerfonden #redeye #avanza #nordnet #seb #swedbank #nordea #aktiespararna #privataaffarer #vatorsecurities #erikpenserbank #carlsquare

Today, September 7, we acknowledge World Duchenne Muscular Dystrophy Awareness Day and extend our deepest gratitude to the Duchenne community and C-Path’s Duchenne Regulatory Science Consortium members for their tireless dedication to advancing treatments for DMD. To combat the many challenges posed by this disease, D-RSC has created an integrated database of patient-level clinical data from DMD studies, and several drug development tools available to the Duchenne community with permissions. Established by C-Path and Parent Project Muscular Dystrophy, D-RSC, in partnership with CDISC, has also developed standard terminology and authored the Duchenne Muscular Dystrophy Therapeutic Area User Guide and the Cardiac Imaging Supplement currently out for public review until September 30.   We are proud to continue this work and are grateful for the community’s ongoing collaboration. For more information, visit c-path.org/programs/d-rsc. To view the Cardiac Imaging Supplement, visit: https://lnkd.in/g8ujGTsV   Ramona Belfiore-Oshan, Pat Furlong   #CPath #DMDawareness #WDAD2024 #musculardystrophy #raredisease #drugdevelopment #datasharing #globalhealth #collaboration #CDISC

Today marks International Ataxia Awareness Day, and we are proud to share that C-Path's ataxia consortium Executive Director Terina N. Martinez, Ph.D. represented us during today's United Against Ataxia Hill Day, a virtual advocacy event sponsored by the National Ataxia Foundation and the Friedreich's Ataxia Research Alliance (FARA). In this critical event, Dr. Martinez had four scheduled meetings with congressional leaders, including Representatives Seth Moulton, Jim McGovern Congressman, Lori Trahan, and Senator Elizabeth Warren and Senator Edward Markey. The discussions centered around crucial initiatives, from supporting the continued inclusion of hereditary ataxia in defense appropriations, to advocating for robust funding for the The National Institutes of Health and FDA, and advancing pediatric and neurological rare disease therapy development. The Critical Path to Therapeutics for the Ataxias consortium exists to unite experts in ataxia research and drug development, creating regulatory tools and strategies to accelerate therapeutic advancements for these rare neurodegenerative disorders. By leveraging aggregated datasets through C-Path’s FDA-funded Rare Disease Cures Accelerator-Data and Analytics Platform, CPTA enhances research insights, facilitating data-driven decisions that optimize the development of treatments for inherited ataxias. We are thankful for opportunities like this to engage with policymakers and make a tangible difference in the lives of those affected by ataxias. Learn more about CPTA, here: https://lnkd.in/gEXy_Ep2 #CPath #Ataxia #NIH #FDA #AtaxiaAwareness #NAF #FARA #drugdevelopment #advocacy #hillday #datasharing #collaboration

PrevEp CSO Dr. Wolfgang Löscher is going to present two talks at the 15. European Epilepsy Congress (EEC https://lnkd.in/eCX6Crwj) organized in Rom from September 7-11, 2024: 1.           Treatment of seizures in neonates, something old, something new - New approaches for the treatment of neonatal seizures 2.           The impact of lamotrigine and topiramate in the past 30 years, and their new derivatives - Drug development based on similar mechanisms of action

🔍 Recent research from Shuchi Gulati, MD, MSc, and colleagues found that #everolimus failed to improved #RFS in patients with #nccRCC. 🧠 Learn More:

🚀Phagenesis Secures $42M in Series D Funding🌐 Exciting news as Phagenesis Ltd closes a $42 million Series D round led by EQT Life Sciences. Their cutting-edge #neurostimulation therapy, Phagenyx®, addresses swallowing dysfunction caused by brain injury or ventilation. With recent FDA approval, the focus is on U.S. commercialisation and deepening European penetration. Funds will boost clinical trials, regulatory activities, and R&D for pipeline products. "This investment from a highly experienced international investor syndicate will accelerate access to and adoption of our therapy," said Reinhard Krickl, CEO of Phagenesis. "We will invest in exceptional talent to scale up our customer outreach and to support passionate clinicians who want to bring our therapy to those who need it. Our novel and proven therapy can help the millions of patients every year who suffer from swallowing disorders." Congratulations on this recent invest Reinhard and the wider Phagenesis team! Full Article in the comments. #investment #phagenesis #dysphagia

Congrats to our clinical data manager Sara Anderson (Weigel) who spoke at the 2024 Zelta User Conference hosted by Merative in Boston earlier this month. Sara shared her perspective on how SPR Therapeutics leverages Zelta for post-market, retrospective real-world data collection.    Zelta is a large part of our clinical study process. We use their clinical data management platform to build and house the databases for our clinical trials. When our study sites enter subject data into the system, it provides our clinical and R&D teams with the ability to monitor, compile and analyze the data. The data is then used to develop abstracts, journal articles and symposium presentations. It is a tool that allows us to be true partners with all of our clinical study sites and offer real-world perspectives in data to educate others about peripheral nerve stimulation.   #PNS #SprintPNS #neuromodulation #clinicaltrials #clinicaldata #clinicalstudies #professionaleducation #leadersinmeddevice #meddeviceleaders

Researchers have developed a pioneering once-a-day pill, SPG302, aimed at regenerating nerve cell connections damaged by ALS (amyotrophic lateral sclerosis). This drug, designed by Spinogenix, Inc., has been FDA-approved for ongoing clinical trials and is now being tested on ALS patients. ALS, also known as Lou Gehrig's disease, progressively damages motor neurons controlling muscle movements, leading to fatal muscle control loss. While current treatments only manage symptoms or slow progression, SPG302 offers a novel approach by focusing on synapse regeneration to restore neuron communication. Early-stage trials in Australia demonstrated that the drug is well-tolerated, leading to FDA approval for further trials in the US. This new phase aims to determine whether SPG302 can significantly improve ALS patients' lives by recovering lost motor and cognitive functions. Read more: https://lnkd.in/e3v3CZZA #medical #als #science #drugdevelopment #biology