Social Media
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
Results from the CARTITUDE-1 Study in Patients With Multiple Myeloma
Results from the VERIFY Clinical Trial in Polycythemia Vera
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
Chronic Myeloid Leukemia: Advice to Newly Diagnosed Patients
Chronic Myeloid Leukemia: Treatment
Chronic Myeloid Leukemia: Overview and Landscape
Chronic Myeloid Leukemia: Overview, Treatment Options, and Advice
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
Tenosynovial giant cell tumor (TGCT) is a non-malignant tumor involving the joint synovium, bursae, and tendon sheath.
Learn more at https://checkrare.com/tenosynovial-giant-cell-tumor-tgct/
#CheckRare #RareDisease #TGCT
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 Data From EMBARK Study in Patients With Duchenne Muscular Dystrophy Receiving Gene Therapy
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
Linvoseltamab Combination Therapy in Patients With Relapsed/Refractory Multiple Myeloma
📢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
Results from the CARTITUDE-1 Study in Patients With Multiple Myeloma
CheckRare June 23, 2025 2:28 pm