NORD Breakthrough Summit 2022

Complete Coverage

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…

FDA Announces 19 Grants in Rare Disease Research During NORD Annual Summit

WASHINGTON, DC—Robert Califf, MD, commissioner of the US Food and Drug Administration (FDA), today announced 19 new grants worth more than $38 million over the next 4 years to support clinical trials, natural history studies, and regulatory science tools related to rare diseases. The grants, funded by the FDA’s Orphan Products Grants Program, are aimed…
MG

Phase 2 of a Novel Monoclonal Antibody Trial for MG Has Begun

An ongoing study will assess the efficacy and safety of an antibody designed to lower immunoglobulin G (IgG) levels in patients with myasthenia gravis (MG), according to a poster presented at the National Organization for Rare Disorders Rare Disease and Orphan Products Breakthrough Summit. “The ongoing Vivacity-MG phase 3 study will assess the efficacy, safety,…
Xpovio (Selinexor)

Diagnosis of MG May Be Delayed for Female Patients

Female patients may receive a more delayed diagnosis of myasthenia gravis (MG) and other neurologic disorders than males, according to a poster presented at the National Organization for Rare Disorders 2022 Rare Diseases and Orphan Products Breakthrough Summit. This descriptive study included data from 3104 patients that received a diagnosis of a neurologic disease considered…
woman holding pill and glass of water

HAE Patients Satisfied With Switching From Injectable Therapy to Oral Monotherapy

A new study revealed high levels of satisfaction among patients with hereditary angioedema (HAE) who switched from injectable prophylactic treatments to berotralstat (Orladeyo®) monotherapy. The results of the study are being presented as a poster at the National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit. “Statistically significant improvements were observed in…

Rare Disease Advisor is pleased to offer live coverage of the 2022 Rare Diseases and Orphan Products Breakthrough Summit. This year, NORD is hosting an in-person event that features all community stakeholders back together in-person in Washington, DC. Join your fellow rare disease leaders from patient advocacy groups, government, industry, and academia for exclusive access to fresh insights, compelling connections, and expert resources.

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