Orlando Agrippa, Founder and CEO of Sanius Health, and Oliver Shastri, team lead for sickle cell disease at Pfizer, discuss how social media can foster community for sickle cell disease patients #CheckRare #RareDisease #SickleCellDisease
checkrare.com/social-media-l…

The recent Priority Medicine #PRIME designation by the European Medicines Agency #EMA for GTX-102 for the treatment of Angelman syndrome provides hope for patients and families #CheckRare #RareDisease #AngelmanSyndrome
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Jatin Shah, MD of Sumitomo Pharma, discusses two treatments in Phase 1 trials for myelofibrosis and acute myeloid leukemia, aggressive diseases that impact the body’s normal production of blood cells #CheckRare #RareDisease #Myelofibrosis #MyeloidLeukemia
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Recreational activities — including golfing, gardening or yard work, woodworking and hunting — may be associated with an increased risk of developing amyotrophic lateral sclerosis #ALS #CheckRare #RareDisease
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A number of children with sickle cell anemia #SCA have low rates of receipt of recommended antibiotic prophylaxis and annual transcranial Doppler ultrasound #CheckRare #RareDisease
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John Charlson, MD, Medical College of Wisconsin, discusses synovial sarcomas and the SPEARHEAD-1 clinical trial testing the safety and efficacy of afami-cel in this rare disease #CheckRare #RareDisease #SynovialSarcoma
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Eosinophilic esophagitis is a chronic autoimmune and digestive disorder in which large numbers of eosinophils, a type of white blood cell, are present in the esophagus. #CheckRare #RareDisease #EoE
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Roberto Giugliani, MD, PhD, discusses the RAINBOW clinical trial testing the tolerability and safety of nizubaglustat for Niemann-Pick type C and GM2 gangliosidosis #CheckRare #RareDisease #NiemannPick #GM2
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Aki Ko, Chief Executive Officer and co-founder of Elixirgen Therapeutics, discusses treatment advances in telomere biology disorders #CheckRare #RareDisease #TelomereBiologyDisorders
checkrare.com/treatment-adva…

WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with neutropenia that typically presents in childhood or adolescence
Check out our learning page for more information
#checkrare #WHIMSyndrome
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Aravindhan Veerapandiyan, MD, pediatric neurologist at Arkansas Children’s Hospital, discusses the AFFINITY clinical trial testing the safety and efficacy of RGX-202 for patients with Duchenne muscular dystrophy #DMD #CheckRare #RareDisease
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For patients with polycythemia vera, rusfertide treatment reduces the use of phlebotomy and maintains hematocrit of less than 45 percent, according to a study published in the Feb. 22 issue of the New England Journal of Medicine #CheckRare #RareDisease
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Bilateral Wilms tumors that increase in size during neoadjuvant chemotherapy, especially in younger patients, are most often stromal-predominant. #CheckRare #RareDisease #BilateralWilms
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Katie Abouzahr, MD, Vice President, Autoantibody Portfolio and Maternal Fetal Disease Area Leader at Johnson & Johnson, discusses nipocalimab for the treatment of myasthenia gravis and other autoantibody diseases. #CheckRare #RareDisease #MyastheniaGravis
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The U.S. FDA has approved Abecma (idecabtagene vicleucel) as a personalized CAR T-cell therapy for triple-class exposed relapsed or refractory multiple myeloma #CheckRare #RareDisease #MultipleMyeloma
checkrare.com/fda-approves-a…

For older patients with acute myeloid leukemia #AML undergoing allogeneic hematopoietic cell transplantation (allo-HCT), posttransplant outcomes have improved over time #CheckRare #RareDisease
checkrare.com/improvements-s…

Tuan Vu, MD, Professor of Neurology at University of South Florida, discusses myasthenia gravis and results from the Phase 3 CHAMPION clinical trial, testing the safety and efficacy of ravulizumab. #CheckRare #RareDisease #MyastheniaGravis
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For patients with arterial calcification due to deficiency of CD73 #ACDC , etidronate may slow progression of vascular calcification in the lower extremities. #CheckRare #RareDisease
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Steven Pipe, MD, of the University of Michigan, discusses the results of HAVEN 7, a Phase 3 trial testing the efficacy and safety of emicizumab to prevent bleeding episodes in patients under 12 months of age with hemophilia A #CheckRare #RareDisease
checkrare.com/treating-sever…

Raymond Douglas, MD, PhD, provides an overview of TED, including diagnosis challenges and current and emerging treatments for this rare disease #checkrare #thyroideyedisease
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