Congenital and Genetic Conditions
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 18, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases
Diagnosing Niemann-Pick Type C
Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular… Jan 18, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Skin Conditions
Ipsen Biopharmaceuticals Commitment to Fibrodysplasia Ossificans Progressiva (FOP)
Stephanie Brown, the SVP of rare diseases at… 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 15, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Skin Conditions
Ipsen Biopharmaceuticals Patient-Focused Approach to Rare Diseases
Stephanie Brown, the SVP of rare diseases at… Jan 15, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases
Overview of Niemann-Pick Type C
Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular… Jan 14, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Diagnosis, Musculoskeletal Diseases
Infantile-Onset Facioscapulohumeral Muscular Dystrophy: Mother and Son Share Their Diagnostic Journey
Ally Roets and her son Sam —… Jan 13, 2021| Posted in: Congenital And Genetic Conditions, Neurology/Nervous System Diseases
Huntington Disease – The Worst Disease Known to Humans
Michael Hayden, PhD, Chief Executive Officer of Prilenia Therapeutics,… 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 11, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Congenital And Genetic Conditions, Drug Development, Metabolic Disorders, News