m6pr igf2r double knockout supplies therapeutic sources for mucolipidosis ii and niemann pick c2

Original title: “M6PR/IGF2R double-knockout supplies therapeutic sources for mucolipidosis II and Niemann-Pick C2.”

identification of serum protein biomarkers in individuals with niemann pick disease type c1

Original title: “Identification of serum protein biomarkers in individuals with Niemann-Pick disease, type C1.”

FDA announces cancer drug trials by AstraZeneca and Amgen will be monitored in real time, a test of how to shorten time interval between trial phases. Monitoring trials in real time may help shorten the time interval between trial phases. The agency also asked the public to weigh in on a potential pilot program to work with companies that use AI to enhance safety monitoring and medication dose selections, identify safety signals, and improve patient recruitment in clinical trials. ... AstraZeneca is conducting a Phase 2 trial of its combination therapy for patients with an aggressive form of lymphoma. The trial will take place at the University of Texas MD Anderson Cancer Center and the University of Pennsylvania. Amgen is conducting a Phase 1b trial of its treatment for small cell lung carcinoma. WASHINGTON — The Food and Drug Administration on Tuesday announced efforts to make clinical trials more efficient, starting by reviewing data in real time from trials conducted by AstraZeneca and Amgen.

Original title: “FDA testing speedier drug development with real-time clinical trials | STAT”

Review upcoming clinical trials with data readouts expected in the first half of 2026, providing updates relevant to ongoing research and clinical practice in neurology. The ADEPT-2 study assesses xanomeline/trospium for psychosis in Alzheimer's, potentially introducing a new class of therapies targeting muscarinic receptor agonism. The ELEVATE-PD trial examines IPX203 for Parkinson's, focusing on real-world efficacy and safety in patients with motor complications. REMODEL studies compare remibrutinib and teriflunomide in relapsing multiple sclerosis, with primary endpoints including annualized relapse rate and disability progression.SHOW MORE · Review upcoming clinical trials with data readouts expected in the first half of 2026, providing updates relevant to ongoing research and clinical practice in neurology. As the field of neurology advances, the early months of 2026 are anticipated to bring important clinical trial readouts that may influence research and care across multiple neurological conditions. These studies span a broad spectrum of disorders and aim to clarify disease mechanisms while evaluating emerging therapeutic approaches. Ranging from novel gene-based strategies to new pharmacologic interventions, the results may inform future treatment paradigms and clinical decision-making. At baseline, patients had a Clinical Dementia Rating-Sum of Boxes (CDR-SB) mean score, the primary end point, of 3.45 (± 1.70). On other secondary outcomes, such as Alzheimer’s Disease Assessment Scale – Cognitive Subscale, 13-item (ADAS-Cog13), patients had a baseline score of 27.75 (± 8.02). Recently, the FDA accepted an updated protocol for Annovis Bio’s · pivotal phase 3 AD study (NCT06709014) testing the therapeutic potential of its investigational agent buntanetap, an orally available small molecule, in AD. The study is designed to evaluate the primary end point of change in the Neuropsychiatric Inventory-Clinician: Hallucinations and Delusions score and the key secondary endpoint of Clinical Global Impression-Severity, with additional assessments on safety and tolerability compared with placebo. In the trial, participants aged 55 to 90 years with possible or probable AD disease will be randomly assigned to either an oral capsule of xanomeline/trospium or an oral capsule of placebo.4 Xanomeline/trospium, which is currently approved for schizophrenia in adults, has the potential to be the first treatment in a new class of pharmacologic therapies targeting agitation and psychosis based on muscarinic receptor agonism.5 Additional study results from the ADEPT program in psychosis associated with AD, including ADEPT-2, ADEPT-1 and ADEPT-4, are expected to be read out by the end of 2026.

Original title: “Key Neurology Trial Readouts to Watch in Early 2026 | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

Catch up on any of the neurology headlines you may have missed in January 2026, compiled into 1 place by the NeurologyLive® team. The coverage includes the latest FDA approvals, new designations, submissions, resubmissions, and clinical trial initiations and holds. Click the read more buttons for more details and information about each update. On January 5, 2026, the FDA approved ScinoPharm Taiwan’s glatiramer acetate (GA) injection as a treatment for relapsing multiple sclerosis (MS), making it the first complex injectable generic approval for this therapy.1 That guidance allows waiving traditional clinical bioequivalence trials if the generic demonstrates tight structural and compositional matching, as well as in vitro and in vivo functional similarity. On January 6, 2026, the FDA granted breakthrough therapy designation to Alkermes’ alixorexton, an investigational oral, selective orexin 2 receptor agonist, for the treatment of narcolepsy type 1 (NT1), using positive phase 1 and phase 2 data, including the phase 2 VIBRANCE-1 trial (NCT06358950), to support the decision. Months after the FDA issued a complete response letter (CRL) to the new drug application (NDA) for Sentynl Therapeutics’ copper histidinate, on January 12, 2026, the agency approved the agent as the first treatment for pediatric patients with Menkes disease, a rare genetic neurodegenerative disorder.3 Marketed as Zycubo, the therapy is a subcutaneous copper replacement treatment that delivers copper in a form designed to bypass impaired intestinal absorption and support systemic utilization of the mineral. "This milestone represents the culmination of decades of research into better understanding and ultimately finding an effective treatment for Menkes disease," Stephen Kaler, MD, a clinical genetics and genomics specialist, and professor of pediatrics at the Columbia University Medical Center, said in a statement.3 "Increased awareness of Menkes disease and rapid testing upon suspicion are critical, as beginning copper histidinate therapy in affected neonates has been shown to reduce symptoms and prolong life."

Original title: “FDA Action Update, January 2026: Approvals, Acceptance, and Clearance | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”