Rare Disease
Latest News
FDA Issues CRL for Vatiquinone in Friedreich Ataxia, Requests More Efficacy Data
Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA
FDA Approves Dordaviprone as First and Only Treatment for Aggressive Form of Glioma
5 Rare Diseases That Now Have Their First FDA-Approved Treatments
How Fitusiran Changes the Treatment Landscape in Hemophilia: Guy Young, MD
Garadacimab, Sebetralstat Were Both Safe to Use in Clinical Trials: Timothy Craig, DO
Combination of Garadacimab, Sebetralstat Can Help Patients With HAE: Timothy Craig, DO
Tiara Green Calls for Comprehensive Support for Rare and Chronic Diseases
Dr Sudipto Mukherjee: Rethinking Clinical Management for Patients With ISM
Dr Sudipto Mukherjee Discusses Survival Outcomes Data in US Patients With ISM
Expanding Horizons: How Every Cure Plans to Increase Rare Disease Drug Access While Reducing Trial Costs
Dr Ruben Mesa on Diversity and Inclusion in Clinical Trials for Rare Diseases
Dr Jeffrey Sippel: Medicare Advantage NIV Denials Take Time From Patients With ALS
Expanding Horizons: Launching a Rare Disease Nonprofit Amid Government Funding Challenges
Dr Rick Chapman Discusses Key Insights From the Center’s Rare Disease Project
Oncology Onward: A Conversation With Dr Shereef Elnahal, Under Secretary for Health
Exploring Payer Coverage Decisions Following FDA Novel Drug Approvals
Huntington Disease Community Regroups After Trial Failures
Personal and Professional: One Doctor's Experience Living With and Treating Cancer
Podcast: This Week in Managed Care - Bipartisan ACA Proposals, and Other Health News
More News
Interventions Needed to Increase DMT Uptake in Sickle Cell Disease
Dr Jeffrey Sippel: The Impact of Insurance Denials for Patients With ALS
Odevixibat Safe for Alagille Syndrome Based on Hepatic Changes
