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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
Inhaled Alprazolam Versus Oral Alprazolam for Acute Seizures
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
FDA Approves Treatment for Transthyretin Amyloid Cardiomyopathy
Investigational Gene Therapy for Parkinson’s Disease with GBA1 Mutations
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
Aromatic L-amino acid decarboxylase #AADC deficiency is a rare genetic disorder that affects the central nervous systems of young patients
Check out our learning page for more information
#checkrare

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the nervous and musculoskeletal systems of young...
checkrare.comThis 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
New Data on Investigational Dravet Syndrome Treatment
Results From the Phase 3 Vivacity-MG3 Study in Myasthenia Gravis
CheckRare 4 hours ago