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AstraZeneca advances scientific leadership in hematology at ASH 2023

Agios Pharmaceuticals to Present Broad Set of Clinical and Translational Data in Rare Blood

– Both Mitapivat Dose Arms Achieved Statistically Significant Hemoglobin Response in Phase 2 Portion of RISE UP Pivotal Study in Sickle Cell Disease, Compared to the Placebo Arm; Data Support

Rigel Announces Five Poster Presentations at the Upcoming 64th American Society of Hematology Annual Meeting and Exposition

Resilient Riley - Johns Hopkins All Children s Hospital

Riley was just a year and a half old when he was diagnosed with a rare genetic condition that affects about 1 in 200,000 people. With the specialized care and teamwork of the Center for Cell and Gene Therapy for Non-Malignant Conditions, Riley, now 5, is back home with his siblings and growing stronger every day.

Novel Stem Cell Transplant Approach Helps Cure Rare Condition - Johns Hopkins All Children s Hospital

Jocelyn was born with a rare primary immunodeficiency disorder that often meant she couldn’t participate in the same things as other kids her age. But that has changed with a new stem cell transplant approach at Johns Hopkins All Children’s that allowed her brother to be a half-matched donor.

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