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New Drug Shows Promise in Wolfram Syndrome

🧬 AMX0035 Shows Sustained Benefit in Wolfram Syndrome at 48 Weeks Amylyx Pharmaceuticals has reported positive long-term results from its Phase 2 HELIOS trial evaluating AMX0035 in individuals with Wolfram syndrome, a rare, progressive neurodegenerative disease. At 48 weeks, the treatment continued to demonstrate improvements or stabilization across multiple disease domains, suggesting durable therapeutic benefit. 🔍 Key Highlights Pancreatic Function: C-peptide response—a marker of residual...

💤 Oral Therapy AD109 Shows Promise in Treating Obstructive Sleep Apnea A recent phase 3 clinical trial has shown that AD109, an investigational oral therapy, significantly reduces the severity of obstructive sleep apnea (OSA) over a six-month period. The SynAIRgy study focused on adults with mild to severe OSA who were either intolerant of or declined CPAP therapy. 🔍 Key Findings Marked Reduction in AHI: Participants receiving AD109 experienced a 55.6% decrease in apnea-hypopnea index (AHI),...

🧠 Semaglutide’s Appetite-Suppressing Effects Traced to Brainstem Neurons A new study in Cell Metabolism reveals how semaglutide, a GLP-1 receptor agonist, suppresses appetite and drives weight loss through a specific set of neurons in the brainstem. The findings help explain the neural basis behind one of the most effective anti-obesity medications currently in clinical use. 🔍 Key Highlights Brainstem Circuit Activation: Researchers identified a population of neurons expressing the Adcyap1...

🧬 Verily Secures $14.7M Grant to Advance Personalized Parkinson’s Research Verily has been awarded a $14.7 million grant from The Michael J. Fox Foundation to enhance its Personalized Parkinson’s Project (PPP)—a large-scale initiative focused on integrating digital, biological, and clinical data to transform Parkinson’s disease research and care. 🔍 Key Highlights Comprehensive Data Collection: The expanded PPP will combine genomic, proteomic, metabolomic, imaging, and biospecimen analyses...

⚡ Neuromodulation Device Demonstrates Long-Term Efficacy in Drug-Resistant Focal Epilepsy A recent study highlights the sustained effectiveness of a neuromodulation device in treating individuals with drug-resistant focal epilepsy over a three-year period. The device, designed to deliver targeted electrical stimulation to specific brain regions, has shown promise in reducing seizure frequency among patients unresponsive to traditional pharmacological treatments. 🔍 Key Findings: Significant...

🧠 Closed-Loop Vagus Nerve Stimulation Boosts Recovery in Chronic Spinal Cord Injury A breakthrough neuromodulation strategy has shown that closed-loop vagus nerve stimulation (CLV), when paired with task-specific physical therapy, can significantly enhance motor recovery in individuals with chronic, incomplete cervical spinal cord injuries. 🔬 Key Highlights Precision-Timed Stimulation:A miniaturized implant delivers vagus nerve stimulation only during successful movements, reinforcing neural...

🧠 FDA Clears First Blood-Based Diagnostic Test for Alzheimer’s Pathology Fujirebio has received FDA 510(k) clearance for its Lumipulse® G pTau 217/β-Amyloid 1-42 Plasma Ratio test—the first blood-based in vitro diagnostic (IVD) test in the U.S. to aid in identifying amyloid pathology associated with Alzheimer’s disease. This test offers a less invasive, more accessible alternative to PET imaging or lumbar puncture for diagnosing Alzheimer’s-related changes. 🔬 Key Highlights Biomarker...

📱 FDA Authorizes CT-132: First Prescription Digital Therapeutic for Episodic Migraine Prevention Click Therapeutics has received FDA marketing authorization for CT-132, the first prescription digital therapeutic (PDT) approved for the preventive treatment of episodic migraine in adults. Delivered via a smartphone app, CT-132 offers a non-pharmacologic, adjunctive option to existing migraine treatments. Key Highlights: Regulatory Milestone: Approved through the FDA’s De Novo pathway, CT-132 is...

Psilocybin Therapy Shows Promise for Mood Dysfunction in Parkinson’s Disease A recent open-label pilot study published in Neuropsychopharmacology explored the safety and potential efficacy of psilocybin-assisted therapy for treating depression and anxiety in individuals with Parkinson’s disease (PD). This research marks the first investigation of psilocybin's effects in a neurodegenerative disease population. Study Overview: Twelve participants with mild to moderate PD and comorbid depression...

🧠 FDA Clears Precision Neuroscience’s High-Resolution Cortical Interface Precision Neuroscience has received FDA 510(k) clearance for its Layer 7 Cortical Interface, a high-resolution electrode array designed to record, monitor, and stimulate electrical activity on the brain's surface. This device, a core component of the company's developing wireless brain–computer interface (BCI) system, is now authorized for commercial use with implantation durations of up to 30 days. Extended...