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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
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
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
Real-World Evidence Supporting ANX005 for Patients With Guillain-Barré Syndrome
Marina Kremyanskaya, MD, PhD, Icahn School of Medicine at Mount Sinai, discusses the development of gene silencer divesiran for treatment of polycythemia vera (PV).
https://checkrare.com/development-of-gene-silencer-for-treatment-of-polycythemia-vera/
📣 Stay up to date on the most recent FDA approvals and PDUFA dates in the rare disease space with our 2025 Orphan Drugs webpage!
https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/
#CheckRare #RareDiseases #PDUFADates #FDAApproval
This CME program, hosted by John Kuruvilla, MD, explores best practices for discussing possible clinical trial participation with patients who have hematologic malignancies.
#CheckRare #CME
This CME program, hosted by Richard J. Nowak, MD, MS, explains the role of neonatal fragment crystallizable receptor (FcRn) in myasthenia gravis (MG) and how treatments that target FcRn are being used to manage patients with MG.
#CheckRare #CME #MyastheniaGravis
This CME program, developed by Howard Trachtman, MD, and Carla M. Nester, MD, addresses the complexities involved in diagnosing, treating, and managing patients with various complement-mediated kidney disorders, such as C3 glomerulopathy and atypical hemolytic uremic syndrome (aHUS).
...#CheckRare #CME #RareDisease #KidneyDisorders
CheckRare is delighted to once again be covering #ASH2024
#CheckRare #RareDisease
In honor of PKU Awareness Day today, PTC Therapeutics is proud to join the #PKU community in raising awareness about phenylketonuria and the unique challenges of managing the condition. Together, we aim to reimagine a future where those living with PKU can live more freely. Sign up today to receive... updates about the future of PKU management. Learn more: www.reimaginepku.com
New Clinical Trial For Late Onset Pompe Disease Begins
Initiation of Biomarker Cohort in Clinical Trial for Facioscapulohumeral Muscular Dystrophy
The CheckRare team is at #GRIDS2024
Stay tuned for coverage from the meeting!
#CheckRare #RareDisease
Vascular Ehlers-Danlos Syndrome: Diagnosis and Advocacy
Results From the Phase 3 Vivacity-MG3 Study in Myasthenia Gravis
CheckRare 4 hours ago