Bob Herpen

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.

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.

1 week ago | healio.com | Bob Herpen

Key takeaways: The approval applies to certain patients with myasthenia gravis or chronic inflammatory demyelinating polyneuropathy. An expert said the new option allows patients “to manage their condition more independently.”The FDA has approved a subcutaneous self-injectable for adults with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive and adults with chronic inflammatory demyelinating polyneuropathy.

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 ...

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.