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1 week ago | 
healio.com | Justin Cooper |Shenaz Bagha
Research into Sjögren’s disease — both analyzing the condition itself and testing potential treatments — has come a long way in the past decade. “Fifteen years ago, we had no clinical trials, no pharma interest in Sjögren’s, and we were starting to see lupus trials fail one after the other,” Kathy M. Hammitt, vice president of medical and scientific affairs at the Sjögren’s Foundation, told Healio.
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1 week ago | 
healio.com | Bob Herpen |Shenaz Bagha
Key takeaways: RGX-202 was safe and well-tolerated in all 12 patients at two different doses with no serious adverse events. The treatment led to timed function test improvements vs. a natural history cohort. SAN DIEGO — Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory boys with Duchenne muscular dystrophy, interim data show.
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1 week ago | 
healio.com | Justin Cooper |Shenaz Bagha
Read more Key takeaways:Patients misdiagnosed as psychosomatic/psychiatric reported long-term distrust in clinicians and reduced mental health and self-worth. Past misdiagnoses should be discussed and empathized with. Long-term impacts to mental health and trust in health care providers can result from the misdiagnosis of rheumatic diseases as psychosomatic or psychiatric conditions, according to data published in Rheumatology.
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1 week ago | 
healio.com | Bob Herpen |Shenaz Bagha
Key takeaways: Assessing apolipoprotein E genotypes and baseline MRI data are key to identifying higher risk patients. ARIA presence was highest in patients treated with donanemab across all included clinical trials. SAN DIEGO — The risk for amyloid-related imaging abnormalities in early-stage Alzheimer’s disease is primarily driven by the apolipoprotein E e4 genotype, as well as hemorrhaging present at baseline, according to a speaker.
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1 week ago | 
healio.com | Bob Herpen |Shenaz Bagha
Key takeaways: Treatment with leriglitazone showed disease arrest in seven of 20 patients. The treatment was generally well-tolerated, with adverse events being mild to moderate. SAN DIEGO —Leriglitazone appeared to stop disease progress both clinically and via imaging in a small cohort of boys with cerebral adrenoleukodystrophy, or cALD, at 96 weeks, according to a presenter. “Cerebral adrenoleukodystrophy ... occurs rapidly in childhood and more than 90% of these boys ...
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2 weeks ago | 
healio.com | Bob Herpen |Shenaz Bagha
Key takeaways: A majority of caregivers reported improvements in patients’ speech, alertness and social interactions. Outcomes also improved with respect to quality-of-life metrics and gastrointestinal issues. SAN DIEGO — Among individuals with Rett syndrome, treatment with trofinetide was linked to improvements in caregiver-reported outcomes by 9 months, according to a speaker at the American Academy of Neurology Annual Meeting.
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2 weeks ago | 
healio.com | Moira Mahoney |Shenaz Bagha
Key takeaways: Therabot users experienced reduction in symptoms for major depressive disorder, generalized anxiety disorder and feeding and eating disorders. Participants rated the Therabot app similarly to a human therapist. Adults showed a significant reduction in their clinical-level mental health symptoms with unrestricted access to a generative AI-powered chatbot designed for mental health treatment, according to results of a randomized trial.
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2 weeks ago | 
healio.com | Stephanie Viguers |Shenaz Bagha
Key takeaways: In TEMPO-1, fixed 5 mg and 15 mg doses of tavapadon improved outcomes in patients with Parkinson’s disease. TEMPO-2 showed that a flexible dose was also associated with significant improvements. SAN DIEGO — Fixed and flexible doses of tavapadon significantly improved motor function compared with placebo in patients with Parkinson’s disease, according to a pair of phase 3 trials presented at the American Academy of Neurology annual meeting.
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2 weeks ago | 
healio.com | Stephanie Viguers |Shenaz Bagha
Key takeaways: Pridopidine showed promise in patients with Huntington’s disease who were not on antidopaminergic medications. The safety and tolerability of pridopidine was comparable to placebo. SAN DIEGO — Pridopidine had a positive effect on clinical progression, cognition and motor function in patients with Huntington’s disease who were not on antidopaminergic medications, according to a pre-specified analysis of the PROOF-HD trial.
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3 weeks ago | 
healio.com | Bob Herpen |Shenaz Bagha
Key takeaways: U.S. coastal areas with greater levels of marine microplastics had higher rates of disabilities among residents. Specific disabilities included those related to memory, thinking, mobility and independent living. Living along coastal areas in the United States that had higher levels of microplastics in the ocean was strongly associated with an increased risk for disability vs. living in coastal areas with lower levels, preliminary data show.
