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
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:
- Taletrectinib for the treatment of adults with locally advanced or metastatic ROS1-positive NSCLC
- Garadacimab for the treatment of HAE in patients ages 12 years and older
Learn more at ...https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/
#CheckRare #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
Implications of Nipocalimab Approval on Pediatric Patients With Myasthenia Gravis
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
Results from the CARTITUDE-1 Study in Patients With Multiple Myeloma
CheckRare June 23, 2025 2:28 pm