Rare Disease
Latest News
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
Protein Level Correlates With Pediatric SMA Severity, Treatment Response
Muscle-Directed Therapy Apitegromab Meets Primary End Point in Phase 3 SAPPHIRE Trial of SMA
Exploring the Implementation of Quality Care Programs for Patients with Myeloproliferative Neoplasms
Bruce Feinberg, DO; Kathy Oubre, Michael Reff, RPh, MBA; Jamile M. Shammo, MD; and Ruben Mesa, MD, discuss the incorporation of quality care programs for improved disease management and patient care for MPNs.
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
