Featured

Updates in Refractory Chronic Cough From ERS 2025

Jacky Smith, PhD, Chair of Respiratory Medicine at the University of Manchester, discusses topline results from the phase 2a RIVER trial for patients with refractory chronic cough (RCC). 4 days ago

Zopapogene Imadenovec in Treating Patients With Recurrent Respiratory Papillomatosis

Clint Allen, MD, Senior Investigator, and Scott Norberg, DO, Associate Research Physician at Center for Cancer Research at the National Cancer Institute, discuss results from a study testing the safety and efficacy of zolapogene imadenovac to treat patients with recurrent respiratory papillomatosis (RRP). 5 days ago

Survey Finds Wide-Ranging Impact of X-Linked Hypophosphatemia (XLH) on Patient Experiences

Findings highlight the burden of XLH on personal finances and out-of-pocket costs, overall health—physical, mental, and social—and access to expert care. 1 month ago

Naxitamab Combination Therapy for Patients With Neuroblastoma

Javier Oesterheld, MD, Division Chief of the Cancer and Blood Disorders Program at Levine Children’s Hospital and Founder and Executive Director of the ARISE Cancer Consortium, discusses naxitamab combination therapy for the treatment of patients with relapsed/refractory high-risk neuroblastoma. 1 month ago
PROBE Staging Tool

New Staging Tool Available for Healthcare Providers Treating Patients With CTCL

Pamela Blair Allen, MD, MSc, Hematologist, discusses the new PROBE staging tool for cutaneous T-cell lymphoma (CTCL). 2 months ago

Panel Discussion: The High Cost of Rare Diseases  

Dr. Joni Rutter of NCATS and Annie Kennedy of the EveryLife Foundation discuss four recent studies estimating the direct and indirect costs of rare diseases in the United States.  2 months ago

Chronic Myeloid Leukemia: Diagnosis and Treatment

Michael Mauro, MD, Director of the Chronic Myeloid Leukemia Program at Memorial Sloan Kettering Cancer Center, discusses the diagnosis and treatment of chronic myeloid leukemia (CML). 3 months ago
Results for Avapritinib

Promising Results for Avapritinib in Observational Trial of Patients With Indolent Systemic Mastocytosis

Researchers have confirmed promising results for avapritinib in an observational study with patients with uncontrolled symptoms of ISM. 3 months ago
Chronic Myeloid Leukemia Overview

Chronic Myeloid Leukemia: Overview, Treatment Options, and Advice

Jorge Cortes, MD, Director of the Georgia Cancer Center, provides an overview of chronic myeloid leukemia (CML), discusses treatment options, and offers advice to newly diagnosed patients. 4 months ago

Priority Review Voucher Tracking

The Priority Review Voucher (PRV) program incentivizes the development of drugs for rare pediatric and tropical diseases. 4 months ago
A New Test for Patient-Reporting of Mastocytosis Control

A New Test for Patient-Reporting of Mastocytosis Control

A team of allergists, immunologists, and dermatologists from Germany and the United States sought to test a new patient-reported outcomes measure for assessing mastocytosis disease control. 4 months ago

Progressive Familial Intrahepatic Cholestasis (PFIC): Diagnosing, Treating, Monitoring

Drs. Patrick McKiernan and Nadia Ovchinsky discuss the recently published guidance on best practices to diagnose, treat, and monitor patients with progressive familial intrahepatic cholestasis (PFIC). 5 months ago

Consider Rare: Suspecting and Diagnosing Fibrodysplasia Ossificans Progressiva (FOP)

This educational webinar on fibrodysplasia ossificans progressiva (FOP), hosted by Ellen Elias, MD and Christiaan Scott, MD, examines best practices to suspect and diagnose this ultra-rare condition. 5 months ago
Neuroblastoma: Treatment

Neuroblastoma: Overview, Curie Scores, and Treatment Options

Greg Yanik, MD, Clinical Professor at University of Michigan's C.S. Mott Children's Hospital, provides an overview of this rare childhood cancer, goals of therapy, using the Curie score, and immunotherapy treatments. 6 months ago
Neuroblastoma: EVAN Foundation

Neuroblastoma: Evan Foundation

Gavin Lindberg details his family’s experience with neuroblastoma, provides advice to families, and discusses the EVAN Foundation. 6 months ago
Neuroblastoma

Neuroblastoma

Neuroblastoma is a rare childhood cancer, but it is the most common extracranial solid tumor in children. 7 months ago
Improving Health Equity in HAE

Improving Health Equity in Hereditary Angioedema (HAE): A Panel Discussion

This panel discussion by three clinical research leaders in HAE, Drs. Aleena Banerji, Timothy Craig, and Marc Riedl, provide an overview of the discrepancies in care observed in certain patient populations, as well as a discussion on best practices to reduce those inequalities moving forward. 8 months ago
WHIM Syndrome Overview

WHIM Syndrome: Overview, Diagnosis, and Management

Jean Donadieu, MD Service d'Hémato-Oncologie Pédiatrique, Hopital, Paris, provides an overview of WHIM syndrome. 8 months ago