Emerging therapeutics may improve outcomes in pediatric patients with rare disorders

SEATTLE — Clinicians should consider disease-modifying therapeutics when treating patients with rare pediatric disorders, according to an expert at the 2022 American Academy of Neurology annual meeting.
“I want people to think about the rationale behind genetic-based therapies,” Lisa Emrick, MD, assistant professor of neurology and developmental neuroscience at Baylor College of Medicine, told attendees. “But I also want everyone ... to think about ways that, even in your clinic, you can help these patients with varied genetic disorders.”
Emrick based the

SEATTLE — Clinicians should consider disease-modifying therapeutics when treating patients with rare pediatric disorders, according to an expert at the 2022 American Academy of Neurology annual meeting.
“I want people to think about the rationale behind genetic-based therapies,” Lisa Emrick, MD, assistant professor of neurology and developmental neuroscience at Baylor College of Medicine, told attendees. “But I also want everyone … to think about ways that, even in your clinic, you can help these patients with varied genetic disorders.”
Emrick based the