ACMG - American College of Medical Genetics and Genomics’ Post

Alpha Cognition, Inc. has reported its financial results and updated progress for Q1 2024. The company's lead drug, ALPHA-1062, is currently under FDA review for mild-to-moderate Alzheimer's disease. If approved, this unique treatment option is expected to make a meaningful difference in the lives of Alzheimer's patients. ALPHA-1062 is a patented acetylcholinesterase inhibitor with minimal gastrointestinal side effects and positive cognitive effects. Alpha Cognition is dedicated to developing treatments for neurodegenerative diseases with limited options. Learn more about ALPHA-1062's potential impact on the lives of Alzheimer's patients from the link below. https://lnkd.in/dAtiHSDr Don Kalkofen Michael Mcfadden

🔊 Exciting news in the field of neuromuscular disorders! The FDA has approved Duvyzat (givinostat), the first nonsteroidal oral medication for treating Duchenne Muscular Dystrophy (DMD) in patients aged six and older. This groundbreaking drug, which reduces inflammation and muscle loss, offers a new treatment option for all genetic variants of DMD. This rare neurological disorder causes progressive muscle weakness. ✔ 💊 The approval of Duvyzat is a significant step forward in the fight against DMD, providing hope for those affected by this devastating disease. The drug's efficacy was proven in an 18-month Ph3 study, showing a statistically significant decline in muscle function compared to the placebo. 💪 🏷 While this is a significant breakthrough, it's important to note that Duvyzat does come with potential side effects and specific prescribing information. 🔦 🚀 Congratulations to ITALFARMACO S.P.A. on this achievement and their commitment to advancing therapies for DMD. 👏 👉 For more details, click on the link - https://lnkd.in/g8cM7e_s #FDA #DMD #medicalinnovation #healthcarenews

Following on my post from yesterday, here’s more progress on cannabinoid drug development on the other spectrum of patients, older adults with neurodegeneration. Here the researchers at the Salk Institute are working on CBN and designing more effective and targeted drugs to lessen nerve cell death and damage to help patients heal and improve symptoms. https://lnkd.in/g6bwx4an

Nearly half of the deaths related to traumatic injury occur from hemorrhagic shock and sepsis, yet advancements in treating shock have been limited. A new study by researchers from Virginia Commonwealth University School of Medicine, used a preclinical model to test the hypothesis that poor oxygenation of the gut and subsequent ischemia-induced metabolic cell swelling could be reversed by a PEG-20k based cell impermeant IV solution. This treatment resulted in reduced indices of shock, increased survival time and preserved gut barrier function. Read their article here: https://bit.ly/48ZOfRE. #pharmacology

Following on my post from yesterday, here’s more progress on cannabinoid drug development on the other spectrum of patients, older adults with neurodegeneration. Here the researchers at the Salk Institute are working on CBN and designing more effective and targeted drugs to lessen nerve cell death and damage to help patients heal and improve symptoms. https://lnkd.in/g6bwx4an

Today we presented exciting preclinical data on our development candidate MRT-8102, demonstrating that it’s a potent, selective, and durable molecular glue degrader (MGD) of NEK7.    The data provide preclinical proof of concept that a NEK7 MGD leads to inhibition of the NLRP3 inflammasome and IL-1 release to reduce the effects of inflammation, supporting the potential to address central and peripheral inflammatory disorders. https://bit.ly/4d18243

Exciting news from NLS Pharmaceutics! We´ve partnered with Aexon Labs on a groundbreaking patent application for dual orexin receptor agonists.These innovative compounds target narcolepsy and neurodegenerative diseases like Parkinson´s, promising safer and more effective treatments. Read the full article here: rb.gy/g9e468 #nlspharmaceutics #cns #narcolepsy #narcolepsyresearch #parkinson

#AnavexLifeSciences (NASDAQ:AVXL) shares plunged this week on mixed results from its Rett Syndrome clinical study but analysts at Dawson James still see the stock as a worthwhile investment.  The company reported topline results from its Phase 2/3 EXCELLENCE study in pediatric Rett Syndrome which had enrolled 92 patients aged from five to 17 years. Rett Syndrome is a rare genetic mutation affecting brain development usually in girls for which there is no cure but symptoms can be managed with medication, physio- and speech therapy and nutritional support.  After 12 weeks, the company’s therapeutic ANAVEX2-73 resulted in an improvement in the key co-primary endpoint Rett Syndrome Behavior Questionnaire, a detailed 45-item questionnaire for assessing the disease’s characteristics completed by the patient’s caregivers. More at #Proactive #ProactiveInvestors #NASDAQ #AVXL http://ow.ly/Tb561057RMe

For #WorldAlzheimersDay tomorrow, we stand with Alzheimer's Disease International during #WorldAlzheimersMonth to raise awareness and continue making progress in addressing this disease. This year’s taglines include “Time to act on dementia, Time to act on Alzheimer’s.” #Alzheimer’s disease is a degenerative brain disease that affects memory loss and cognitive ability. At JCR Pharmaceuticals, we are looking to build partnerships with companies using our proprietary platform technology, J-Brain Cargo®, to enable a partner’s biotherapeutic to cross the blood-brain barrier and address neurodegenerative conditions like Alzheimer’s disease with next-generation treatments. To learn more about #WorldAlzheimersDay, visit: https://bit.ly/3ArPKvw. #JCRPharma #Neurodegeneration #CNS #BloodBrainBarrier #Alzheimer's

In a first-of-its-kind study, peptide receptor radionuclide therapy, or PRRT, has been deemed the most effective way to treat neuroendocrine tumors (NETs) compared to everolimus. When managing tumors that have as high a fatality rate as NETs, it's important for both doctors and patients to understand the most effective, least toxic treatments. Read the entire article in this issue of JNM: https://lnkd.in/e25fsRYF