Rare Disease
Latest News
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
FDA Approves Mirdametinib for Neurofibromatosis Type 1 With Plexiform Neurofibromas
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
