CLINICAL PERSPECTIVES +++++ +++++

CLINICAL PERSPECTIVES Sotatercept for Pulmonary Arterial Hypertension Within the First Year After Diagnosis https://checkrare.com/sotatercept-for-pulmonary-arterial-hypertension-within-the-first-year-after-diagnosis/ A recent study, published in the New England Journal of Medicine, looked at the effects of sotatercept for pulmonary arterial hypertension (PAH) within the first year after diagnosis. Effects of Vutrisiran on Cardiac Structure and Function in Patients With Transthyretin Amyloidosis With Cardiomyopathy https://checkrare.com/effects-of-vutrisiran-on-cardiac-structure-and-function-in-patients-with-transthyretin-amyloidosis-with-cardiomyopathy/ A recent study, published in Nature Medicine, looked at the effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) through a secondary analysis of the HELIOS-B clinical trial. Rilzabrutinib Approval for Adult Patients With Immune Thrombocytopenia https://checkrare.com/rilzabrutinib-approval-for-adult-patients-with-immune-thrombocytopenia/ Amit Mehta, MD, Medical Oncologist, discusses data that led to the approval of Wayrilz (rilzabrutinib) for adult patients with immune thrombocytopenia (ITP). Long-Term Data From the AQUARIUS Study of Avacopan in Patients With GPA and MPA Naomi Patel, MD, MPH, Rheumatologist at Massachusetts General Hospital, discusses long-term data from the AQUARIUS study of avacopan in patients with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). https://checkrare.com/long-term-data-from-the-aquarius-study-of-avacopan-in-patients-with-gpa-and-mpa/ Symptom and Treatment Burden in Fabry Disease https://checkrare.com/symptom-and-treatment-burden-in-fabry-disease/ Jack Johnson, Co-Founder and Executive Director of FSIG, discusses symptom and treatment burden in Fabry disease. Recordati Rare Disease Initiatives https://checkrare.com/recordati-rare-disease-initiatives/ Mohamed Ladha, President and General Manager for Recordati Rare Diseases North America, discusses the company’s rare disease initiatives. Marfan Syndrome: Diagnosis and Management https://checkrare.com/marfan-syndrome-diagnosis-and-management/ David Liang, MD, PhD, Cardiologist at Hoag Hospital, discusses the diagnosis and management of Marfan syndrome. Evolving Policy Landscapes for Rare Disease Access https://checkrare.com/evolving-policy-landscapes-for-rare-disease-access/ Deb Jennings, Head of North America Patient Services Operations at Kyowa Kirin, discusses evolving policy landscapes for rare disease access. The Role of Mental Health in Rare Disease Patient Outcomes https://checkrare.com/the-role-of-mental-health-in-rare-disease-patient-outcomes/ Sumira Riaz, PhD, Health Psychologist & Patient Engagement Consultant at Unboxed Psychology, discusses the role of mental health in rare disease patient outcomes. Disease Severity and Progression in Patients With ENPP1 Deficiency https://checkrare.com/disease-severity-and-progression-in-patients-with-enpp1-deficiency/ Matt Winton, PhD, Senior Vice President and Chief Operating Officer of Inozyme Pharma, discusses results from an analysis characterizing disease severity and progression in patients with ENPP1 deficiency.