Panel Discussion: The High Cost of Rare Diseases
Dr. Joni Rutter of NCATS and Annie Kennedy of the EveryLife Foundation discuss four recent studies estimating the direct and indirect costs of rare diseases in the United States.
Dr. Joni Rutter of NCATS and Annie Kennedy of the EveryLife Foundation discuss four recent studies estimating the direct and indirect costs of rare diseases in the United States.
Dr. Smith-Whitley, pediatric hematologist at the Children’s Hospital of Philadelphia, discusses the advances in sickle cell disease.
For hematologic malignancy (HM) patients admitted to the ICU, survival is 49 percent at seven days and 21 percent at 12 months.
Incremental cost-effectiveness ratios were $126,000 and $281,000 per QALY for two simulation models from a societal perspective.
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Treatment with NALL for 12 weeks yields better neurological status than placebo among patients with Niemann-Pick disease type C.
Cat Lutz, PhD, MBA, VP at JAX Rare Disease Translational Center, discusses rare disease research’s impact on understanding common diseases.
Bill Grambley explains how his company attacks health inequalities by providing personalized pharmaceutical care with an AI based platform.
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This symposium, led by Ozlem Goker-Alpan, MD of the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) reviews the current state of newborn screening…