Exagamglogene Autotemcel Meets End Points in Severe Sickle Cell Disease, β-Thalassemia
December 7th 2023Two posters set to be presented at the 65th American Society of Hematology Annual Meeting & Exposition met their primary and secondary end points regarding exagamglogene autotemcel therapy for sickle cell disease and β-thalassemia.
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Odevixibat Safe for Alagille Syndrome Based on Hepatic Changes
November 8th 2023Pooled phase 3 data presented at North American Society for Pediatric Gastroenterology, Hepatology & Nutrition 2023 support the benefit-risk profile of the ileal bile acid transport inhibitor in treating the rare liver disease.
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Contributor: How Patients and Caregivers Can Be a Catalyst for Rare Disease Innovation
November 5th 2023Patient input and experiences play a crucial role in advancing rare disease research and therapy development, as they help define the disease, inform clinical trial design, and influence regulators and payers' decisions, ultimately serving as catalysts for innovation in the field.
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Dr Ryan Haumschild Discusses Payer, Provider Perspectives on Prior Authorization in Rare Diseases
September 19th 2023Ryan Haumschild, PharmD, MS, MBA, director of pharmacy at Emory Winship Cancer Institute, shares his unique perspectives as both a provider and payer on the use of prior authorizations (PAs) and "Gold Cards" in patients with rare diseases.
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Catherine Nester Discusses the BeginNGS Tool for Newborn Rare Disease Screening Awareness
August 25th 2023Catherine Nester, RN, vice president of physician and patient strategies at Inozyme Pharma, highlights the importance of newborn screening for rare diseases and the impact of the BeginNGS diagnostic tool.
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Findings Highlight Need for Systematic, Unbiased Approach to Identifying Drug Culprit for SJS/TEN
July 24th 2023The retrospective study of patient hospital data showed that current approaches to identifying a culprit drug often overidentify drugs unlikely to be responsible for Stevens-Johnson syndrome/toxic epidermal necrolysis (SJS/TEN) while potentially missing the actual culprit.
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Dr Franco Locatelli Discusses the Treatment of Patients with cGvHD
July 21st 2023Franco Locatelli, MD, PhD, head of the Department of Pediatric Hematology and Oncology at IRCCS Bambino Gesu Children’s Hospital in Rome, discusses the treatment of both adult and pediatric patients with chronic graft versus host disease (cGvHD).
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Dr Franco Locatelli: Assessing Ruxolitinib Treatment in Pediatric Patients With cGvHD
July 14th 2023Franco Locatelli, MD, PhD, a professor and hematologist from Italy, discusses results from the the phase 2 REACH 5 studies assessing the use of ruxolitinib in pediatric patients with chronic graft versus host disease (cGvHD).
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