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Carsten Utoft Niemann, MD, PhD, Copenhagen University Hospital, discusses results from the GLOW clinical trial in chronic lymphocytic leukemia (CLL).
https://checkrare.com/results-from-the-glow-clinical-trial-in-chronic-lymphocytic-leukemia/
#CheckRare #RareDisease #RareCancer
📢CME Program: Jean Elwing, MD, of the University of Cincinnati College of Medicine provides an overview of the latest clinical research about PAH presented at CHEST 2024.
https://checkrare.com/learning/p-pah-clinical-research-highlights-chest-2024/
#CheckRare #RareDisease ...#RareLung #CMEProgram
FDA Approval: The U.S. Food and Drug Administration has approved Gomekli (mirdametinib) for the treatment of adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas.
https://checkrare.com/fda-approves-mirdametinib-for-treatment-of-nf1-pn/
...#CheckRare #FDAApproval #RareGenetic
💡Rare Disease Spotlight: Polycythemia Vera
Learn more about this rare disease at https://checkrare.com/?s=polycythemia+vera
#CheckRare #RareDisease #PolycythemiaVera #RareHematology
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
Caplacizumab Combination Therapy in Patients With Acquired Thrombotic Thrombocytopenic Purpura
Cost Comparison of Therapies for Major Surgeries in Patients With Hemophilia A
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 PERSEUS Clinical Trial in Multiple Myeloma
Join Professors João Gonçalves and Paolo Calicaeti in this educational webinar discussing how PEGylation represents a major technological advancement in the development and optimization of therapeutic proteins.
...https://checkrare.com/optimizing-therapeutic-proteins-through-pegylation/
#CheckRare #RareDisease #lysosomal #RareLysosomal
Phase 2 REVIVE Clinical Trial for Polycythemia Vera
Results From Clinical Trials Testing rADAMTS13 for cTTP
Results From the Phase 3 Vivacity-MG3 Study in Myasthenia Gravis
CheckRare February 11, 2025 9:11 am