Social Wall
Robert Mitchell-Thain, chair of the PBC Foundation, discusses how linerixibat reduces itching in patients with primary biliary cholangitis (PBC).
https://checkrare.com/linerixibat-reduces-itching-in-patients-with-pbc/
#CheckRare #RareDisease #RareGastrointestinal #RareAutoimmune
Has your patient been diagnosed with immunoglobulin A nephropathy (IgAN), or Berger’s disease? They may be eligible to take part in a new clinical research study evaluating an investigational treatment option that may help manage and slow disease progression. See if they may be eligible to help ...advance research for the IgAN community. Click here to see if they may qualify.
https://icanclinicalstudy.alexionclinicaltrials.com/?utm_source=CheckRare&utm_medium=Social&utm_content=Awareness&utm_campaign=ICAN
Leticia Orsatti, MD, discusses results from clinical trials examining the safety and efficacy of nerandomilast to treat patients with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF).
...https://checkrare.com/evaluating-nerandomilast-in-patients-with-rare-pulmonary-fibrotic-conditions/
#CheckRare #RareLung #RareDisease
The FDA has recently approved the following.
Stay up to date with upcoming PDUFA dates at https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/
#CheckRare #RareDisease #FDAApprovals
The FDA Priority Review Voucher (PRV) program aims to incentivize the development of drugs for rare pediatric and tropical diseases. PRVs act as a way to skip to the front of the FDA’s line to have a future drug reviewed.
Click here to visit CheckRare’s chart tracking issued PRVs and ...their current status https://checkrare.com/priority-review-voucher/
#CheckRare #RareDisease #PriorityReview
Matt Winton, PhD, Senior Vice President and Chief Operating Officer of Inozyme Pharma, discusses results from an analysis characterizing disease severity and progression in patients with ENPP1 deficiency.
...https://checkrare.com/disease-severity-and-progression-in-patients-with-enpp1-deficiency/
#CheckRare #ENPP1 #RareMetabolic #RareGenetic
NEW: Check out our Rare Disease Recognition and Awareness Days page to stay up to date on the latest rare disease awareness programs.
https://checkrare.com/rare-disease-awareness-days-and-recognition-page/
#CheckRare #RareDisease #RareDiseaseAwareness
Erin Sullivan, Executive Director of Sisters’ Hope Foundation, discusses her family’s experience with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).
https://checkrare.com/a-familys-experience-with-alsp/
#CheckRare #ALSP #RareNeurology ...#RareGenetic
Genetic Testing for WHIM Syndrome: reducing barriers, proper panel analysis, and how to deal with variants of uncertain significance.
Learn more at https://checkrare.com/genetic-testing-for-whim-syndrome/
#CheckRare #WHIMSyndrome #RareGenetic #RareAutoimmune #RareSkin
💡Rare Disease Spotlight: SYNGAP1-Related Disorders
Learn more about this rare disease with our latest article https://checkrare.com/the-current-landscape-of-syngap1-related-disorders/
#CheckRare #RareDisease #SYNGAP1 #RareNeurology #RareGenetic
📢Tomorrow, June 10th at 1:00pm EST
Join us for a Live CME Webinar on Skeletal Involvement in Lysosomal Disorders with Ozlem Goker-Alpan, MD, and Ravi Kamath, MD, PhD.
Click the link to sign up: https://us06web.zoom.us/webinar/register/WN_fCXmUAdMTdysE_HnDniVJw#/registration
...
#CheckRare #LiveCME #RareDisease
Annette Bakker, PhD, Chief Executive Officer of the Children’s Tumor Foundation, discusses repurposing shelved assets for rare diseases.
https://checkrare.com/repurposing-shelved-assets-for-rare-diseases/
#CheckRare #RareDisease
Kimberly LeBlanc, Genetic Counselor, Director of the UDN Coordinating Center at Harvard Medical School, discusses approaching variants of uncertain significance in rare diseases.
https://checkrare.com/the-undiagnosed-disease-network/
#CheckRare #UndiagnosedDiseaseNetwork #UDN ...#RareDisease
Sanjay Ahuja, MD, Pediatric Hematologist, Innovative Hematology and the IHTC, discusses the U.S. FDA approval of Qfitlia (fitusiran) for patients with hemophilia A or B.
https://checkrare.com/phase-3-studies-lead-to-approval-of-fitusiran-for-hemophilia-a-and-b/
#CheckRare ...#RareHematology #Hemophilia
The U.S. Food and Drug Administration has recently approved the following treatments.
Stay up to date with new rare disease treatments approvals at https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/
#CheckRare #FDAApproval #RareDisease
Yuliya Linhares, MD, chief of Lymphoma Services at Miami Cancer Institute, provides an overview of cutaneous T-cell lymphoma #CTCL and discusses some strategies for shortening the diagnostic journey of this rare cancer. #CheckRare #RareDisease
...https://checkrare.com/ctcl-shortening-the-diagnostic-journey-and-starting-treatment-early/
The I CAN Study is now enrolling adults diagnosed with immunoglobulin A nephropathy (IgAN) to evaluate an investigational study treatment option that may improve kidney function and slow the progression of the disease in adults. Click here to learn more.
...https://icanclinicalstudy.alexionclinicaltrials.com/?utm_source=CheckRare&utm_medium=Social&utm_content=Awareness&utm_campaign=ICAN
David Curren, patient advocate and board member for Breath of Hope Rhode Island, discusses his grandson’s diagnostic journey with idiopathic pulmonary hemosiderosis.
https://checkrare.com/a-patients-diagnostic-journey-with-idiopathic-pulmonary-hemosiderosis-iph/
#CheckRare ...#RareLung #IPH
Diagnostic Odyssey With Histiocytosis
Disease Severity and Progression in Patients With ENPP1 Deficiency
Implications of Nipocalimab Approval on Pediatric Patients With Myasthenia Gravis
CheckRare June 13, 2025 12:43 pm