Ashley Lyles

All articles by Ashley Lyles

FDA Approval

Real-World Data and Evidence Help Drive Rare Disease Drug and Device Approval

WASHINGTON, DC—Real-world data and real-world evidence play an integral role in the approval of drugs and devices to manage rare disease, according to a group of experts who spoke at the National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit. The session, which was moderated by John Concato, MD, MS, MPH, an…

newborn

Experts Say Improving Newborn Screening for Rare Disease Requires Collaboration

WASHINGTON, DC—Improving newborn screening for rare diseases requires a long and collaborative process, according to experts who spoke at the National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit. The session, led by Mike Hu, California Volunteer State Ambassador, NORD Rare Action Network, explored current topics dealing with newborn screening. Jeffrey Brosco,…

Clinical trials

Panel: More Progress Needed in Device and Drug Development for Pediatric Patients

WASHINGTON, DC—Clinical trials may not be the way to go in assessing pediatric patients with rare diseases, a speaker said today at a session during the National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit. The session, which was moderated by James Baumberger, MPP, senior director of federal advocacy at the American…

Mental health

The Mental and Emotional Toll of a Rare Cancer Diagnosis

WASHINGTON, DC—A patient, neuropsychologist, and social worker explored the mental and emotional effects of rare cancers on patients, their families, and other loved ones during a session at the National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit. The discussion, facilitated by John Hopper, president of the Fibrolamellar Cancer Foundation, and Jim…

group of doctors

Ethical Rare Disease Research Requires Balance and Collaboration, Experts Say

WASHINGTON, DC—Leaders from industry, government, patient advocacy groups, and academia weighed in on ethical dilemmas associated with genetic modification, equity in access, genetic testing, and more during a session at the National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit. The session, facilitated by Alison Bateman-House, PhD, MPH, an assistant professor of medical…

Diversity research

Panel: Diverse Study Populations Needed in Rare Disease Research

WASHINGTON, DC—Government partners, researchers, and patient advocates discussed strategies for enhancing diversity, equity, and inclusion in rare disease research during a session at the National Organization for Rare Disorders’ (NORD) Rare Disease and Orphan Products Breakthrough Summit. The session was led by Lola Fashoyin-Aje, MD, MPH, deputy director of the division of oncology at the…

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