Treatment
Jan 21, 2021| Posted in: Drug Development, Hematologic Disorders, Metabolic Disorders, News, Treatment
FDA Approves First Ever Treatment for AL Amyloidosis
The U.S. Food and Drug Administration (FDA) has… Jan 21, 2021| Posted in: Congenital And Genetic Conditions, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Treatment
Treatment Options for Niemann-Pick Type C
Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular… Jan 20, 2021| Posted in: Cancers, Hematologic Disorders, Treatment
Results of ECHELON-1 study for Hodgkin Lymphoma
Dr. Anna Sureda, Head of the Onco-Hematology Service… Jan 19, 2021| Posted in: Cancers, Drug Development, Hematologic Disorders, Treatment
Janssen Initiates Rolling Submission to FDA for cilta-cel to Treat Relapsed/Refractory Multiple Myeloma
Mark Wildgust, PhD, Vice President, Oncology Global Media… Jan 18, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Heart Diseases, Treatment
Treatment Options for HoFH
James Underberg, MD, Clinical Assistant Professor of Medicine… Jan 17, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Musculoskeletal Diseases, Treatment
Treating Infantile-Onset Facioscapulohumeral Muscular Dystrophy
Ally Roets and her son Sam — who… Jan 17, 2021| Posted in: Cancers, Hematologic Disorders, Treatment
Overview of ECHELON-1 Study for Hodgkin Lymphoma
Dr. Anna Sureda, Head of the Onco-Hematology Service… Jan 15, 2021| Posted in: Cancers, Drug Development, News, Treatment
FDA Approves Pfizer’s Xalkori for ALCL in Children and Young Adults
The Food and Drug Administration has approved Pfizer's… Jan 12, 2021| Posted in: Congenital And Genetic Conditions, Heart Diseases, Hematologic Disorders, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Skin Conditions, Treatment
Determining the Best Treatment Option for Fabry Disease
Robert Hopkin, MD, Associate Professor of Clinical Pediatrics… Jan 4, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Drug Development, Endocrine Disorders, Gastrointestinal Diseases, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lung Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News, Ophthalmology/Eye Diseases, Skin Conditions, Treatment