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CHEST 2025 IPF Chronic Cough

Updates in IPF Chronic Cough From CHEST 2025

Philip Molyneaux, MD, PhD, a Professor of Interstitial Lung Disease (ILD) at Imperial College London and the Asthma+Lung UK Chair of Respiratory Research at Royal Brompton Hospital, where is also the Director of the UK National Institute for Health and Care Research (NIHR) Clinical Research Facility, discusses the results of the CORAL study.   4 days ago

The Genetics of Epilepsy: The Importance of Identifying Underlying Causes

Accurate diagnosis and treatment of epilepsy challenges the medical community and patients. As many as one third of patients have seizures that are inadequately controlled with available therapies, and treatment response is highly variable. 3 weeks ago
Cutaneous T-Cell Lymphoma (CTCL)

Cutaneous T-Cell Lymphoma

Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders. Cutaneous T-cell lymphoma is a rare group of malignancies, with an incidence of 6.4 cases per 1 million people and represents around 70% of primary cutaneous lymphomas. 4 weeks ago

Watch the DAYBUE® (trofinetide) in Practice Video Series to Hear Expert Insights on the First Treatment for This Rare Disease)

Rett syndrome thought leaders discuss how DAYBUE™ (trofinetide), the first and only FDA-approved treatment for Rett syndrome in adults and pediatric patients 2 years of age and older, plays a role in their patients’ treatment plans, while shedding light on their personal experiences with DAYBUE in their practices. 1 month ago
New Approaches to Neuroblastoma Consortium

Neuroblastoma: New Approaches to Neuroblastoma Consortium

Araz Marachelian, MD, Pediatric Oncologist at Children’s Hospital Los Angeles, discusses the New Approaches to Neuroblastoma (NANT) Consortium and its work in neuroblastoma. 1 month ago
Updates in Refractory Chronic Cough

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). 2 months ago
Treating Recurrent Respiratory Papillomatosis

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). 2 months 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. 3 months ago
Naxitamab Combination Therapy for Neuroblastoma

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. 3 months 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). 3 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.  4 months ago
Chronic Myeloid Leukemia: Diagnosis and Treatment

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). 4 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. 4 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. 5 months ago

Priority Review Voucher Tracking

The Priority Review Voucher (PRV) program incentivizes the development of drugs for rare pediatric and tropical diseases. 5 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. 6 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). 7 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. 7 months ago