CLINICAL PERSPECTIVES: +++

CLINICAL PERSPECTIVES Age-Stratified Clinical Burden of Post-Allogeneic HSCT Complications in Hurler Syndrome (MPS IH) https://checkrare.com/age-stratified-clinical-burden-of-post-allogeneic-hsct-complications-in-hurler-syndrome-mps-ih/ Ali Mohajer, PhD, Qral Group, discusses the age-stratified burden of post-allogeneic hematopoietic stem cell transplantation (post-allo-HSCT) complications in Hurler syndrome (MPS IH). Committee for Medicinal Products for Human Use Recommendation for Sotatercept for the Treatment of Pulmonary Arterial Hypertension https://checkrare.com/committee-for-medicinal-products-for-human-use-recommendation-for-sotatercept-for-the-treatment-of-pulmonary-arterial-hypertension/ Marius Hoeper, MD, Respiratory Physician at Hannover Medical School, Germany, discusses a recent regulatory update by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in the treatment of pulmonary arterial hypertension (PAH). Post Hoc Analysis of Long-Term Efficacy of Migalastat in Females With Fabry Disease https://checkrare.com/post-hoc-analysis-of-long-term-efficacy-of-migalastat-in-females-with-fabry-disease/ A recent post hoc analysis of long-term efficacy of migalastat in females with Fabry disease was published in the Journal of Medical Genetics.