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Results From the Phase 3 Vivacity-MG3 Study in Myasthenia Gravis
Clinical research leaders in HAE provide an overview of the discrepancies in care observed in certain patient populations, as well as a discussion on best practices to reduce those inequalities moving forward.
...https://checkrare.com/improving-health-equity-in-hereditary-angioedema-hae-a-panel-discussion/
#CheckRare #RareDisease #RareGenetic
📹 The CheckRare team spent the week at #WORLDSymposia 2025
Stay tuned for our interviews from the conference!
#CheckRare #RareDisease
Paula Ragan, President and Chief Executive Officer of X4 Pharmaceuticals, discusses the pathway of approval under the European Medicines Agency (EMA) for mavorixafor for patients with WHIM syndrome.
...https://checkrare.com/ema-pathway-to-approval-for-mavorixafor-to-treat-patients-with-whim-syndrome/
#CheckRare #RareDisease #WHIMSyndrome #RareGenetic #RareSkin
Emma Searle, PhD, The Christie NHS Foundation Trust and University of Manchester, discusses bleximenib in combination with chemotherapy to treat patients with acute myeloid leukemia (AML).
https://checkrare.com/chemotherapy-combination-treatment-in-patients-with-acute-myeloid-leukemia/
...
#CheckRare #RareDisease #AML #RareCancer
February 4th is #WorldCancerDay
Although there are over 200 types of rare cancers, only a small number of people are diagnosed with each type. These conditions often present unique challenges in diagnosis, research, and treatment. Today we raise awareness, support research, and advocate ...for better resources.🎗️
To learn more about rare cancers, visit https://checkrare.com/diseases/cancers/
#CheckRare #RareDisease #RareCancer
FDA Approval of Setmelanotide for Patients 2 Years and Older With MC4R Pathway Diseases
Caplacizumab Combination Therapy in Patients With Acquired Thrombotic Thrombocytopenic Purpura
Cost Comparison of Therapies for Major Surgeries in Patients With Hemophilia A
EMPEROR Clinical Trial for Pediatric Patients With Dravet Syndrome
Experiences of Women of Childbearing Age With Epilepsy
FDA Approval: Crinecerfont for Congenital Adrenal Hyperplasia
Inhaled Alprazolam Versus Oral Alprazolam for Acute Seizures
Topline Results of the Honeycomb Trial for GRIN-related Neurodevelopmental Disorders
EMA Pathway to Approval for Mavorixafor to Treat Patients with WHIM Syndrome
Improving Health Equity in Hereditary Angioedema (HAE): A Panel Discussion (Full Program)
Overview: Improving Health Equity in HAE (Chapter 1)
HAE in Rural Communities (Chapter 2)
HAE and Race/Ethnicity (Chapter 3)
Jean Donadieu, MD, Service d’Hémato-Oncologie Pédiatrique, Hopital, Paris, provides an overview of WHIM syndrome.
https://checkrare.com/whim-syndrome-overview-diagnosis-and-magement/
#CheckRare #RareDisease #RareGenetic #RareSkin #WHIM
Results From the Phase 3 Vivacity-MG3 Study in Myasthenia Gravis
CheckRare 5 hours ago