Rare Disease
Latest News
5 Rare Diseases That Now Have Their First FDA-Approved Treatments
FDA Approves Mirdametinib for Neurofibromatosis Type 1 With Plexiform Neurofibromas
Delandistrogene Moxeparvovec Fails to Meet Primary End Point in Phase 3 Study
Shared Decision-Making Is Essential Prior to SMA-Enhancing Treatments
Oral Function Impairment Varies Across Adult Patients With SMA
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 Jeffrey Sippel: The Impact of Insurance Denials for Patients With ALS
Dr Ruben Mesa Shares How Atrium Health Addresses Health Disparities in Rural, Minority Populations
Expanding Horizons: How 2 Organizations Are Tackling Rare Disease Treatment Gaps
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
Odevixibat Safe for Alagille Syndrome Based on Hepatic Changes
