CLINICAL PERSPECTIVES +++ ++++ (videos)

CLINICAL PERSPECTIVES FDA’s Plausible Mechanism Framework and its Effect on Rare Disease Therapy Development https://checkrare.com/fdas-plausible-mechanism-framework-and-its-effect-on-rare-disease-therapy-development/ Stevie Ringel, CEO of Nome Therapeutics, discusses the US Food and Drug Administration’s (FDA) Plausible Mechanism Framework and its effect on rare disease therapy development. Nipocalimab Granted Fast Track Designation in Systemic Lupus Erythematosus https://checkrare.com/nipocalimab-granted-fast-track-designation-in-systemic-lupus-erythematosus/ Richard A. Furie, MD, Chief of the Division of Rheumatology at Northwell Health, discusses nipocalimab for the treatment of systemic lupus erythematosus (SLE). Accelerated Approval of Yuviwel (Navepegritide) for Patients with Achondroplasia https://checkrare.com/accelerated-approval-of-yuviwel-navepegritide-for-patients-with-achondroplasia/ Carlos A. Bacino, MD, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital, discusses the accelerated approval of Yuviwel (navepegritide) for patients with achondroplasia. MESA Extension Study of Sevasemten in Patients With Becker Muscular Dystrophy https://checkrare.com/mesa-extension-study-of-sevasemten-in-patients-with-becker-muscular-dystrophy/ Joanne Donavan, MD, PhD, Chief Medical Officer at Edgewise Therapeutics, discusses the MESA extension study of sevasemten for the treatment of patients with Becker muscular dystrophy (BMD). How The FDA’s Plausible Mechanism Framework Is Accelerating Approval for Osteosarcoma Immunotherapy https://checkrare.com/how-the-fdas-plausible-mechanism-framework-is-accelerating-approval-for-osteosarcoma-immunotherapy/ Paul Romness, CEO of OS Therapies, discusses the Plausible Mechanism Framework draft guidance and how it will affect the approval process of OST-HER2 LM for the treatment of osteosarcoma. First Patient Dosed in FALCON Clinical Trial Evaluating SGT-212 Gene Therapy for Friedreich’s Ataxia https://checkrare.com/first-patient-dosed-in-falcon-clinical-trial-evaluating-sgt-212-gene-therapy-for-friedreichs-ataxia/ Gabriel Brooks, MD, Chief Medical Officer at Solid Biosciences, and Russell Lonser, MD, of The Ohio State University Wexner Medical Center, discuss SGT-212 and the FALCON clinical trial for patients with Friedreich’s ataxia. Phase 1/2 Results of Tividenofusp Alfa in Patients With MPS II https://checkrare.com/phase-1-2-results-of-tividenofusp-alfa-in-patients-with-mps-ii/ Joseph Muenzer, MD, PhD, Pediatric Biochemical Geneticist at University of North Carolina Chapel Hill, discusses results from a phase 1/2 study of tividenofusp alfa in patients with mucopolysaccharidosis II (MPS II).