Congenital and Genetic Conditions
Jul 29, 2020| Posted in: Congenital And Genetic Conditions, Hematologic Disorders, Treatment
Jonathan Sorof, MD, Senior Vice President, Head of…
Expanding Treatment Options for Sickle Cell Disease Patients
Jul 28, 2020| Posted in: Congenital And Genetic Conditions, Diagnosis, Gastrointestinal Diseases, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Skin Conditions
Ozlem Goker-Alpan, MD, provides a summary of research…
Fabry Disease and the Heart
Jul 27, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News
Diego Cadavid, MD of Fulcrum Therapeutics provides an…
Update on the Phase 2 Facioscapulohumeral Muscular Dystrophy Clinical Trial
Jul 24, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Hematologic Disorders, Metabolic Disorders
Federico Grossi, MD, chief medical officer at Apellis…
Paroxysmal Nocturnal Hemoglobinuria (PNH) Explained
Jul 23, 2020| Posted in: Congenital And Genetic Conditions, Neurology/Nervous System Diseases, News, Treatment
Ashish Sagrolikar, Chief Commercial Officer of Zogenix, discusses…
FDA Approval of Fintepia (fenfluramine) to Treat Dravet Syndrome
Jul 21, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News
Diego Cadavid, MD, of Fulcrum Therapeutics summarizes the…
Research Presented at AAN 2020 Show Promise for FSHD Treatment
Jul 20, 2020| Posted in: Autoimmune / Autoinflammatory Disorders, Congenital And Genetic Conditions, Hematologic Disorders, News
Jonathan Sorof, MD, Head of Medical Affairs at…
How COVID-19 Has Affected Sickle Cell Disease Patients
Jul 16, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Kidney And Urinary Diseases, Metabolic Disorders
Marshall Summar, MD, Division Chief, Genetics and Metabolism…
New Study Underway to Treat Propionic Acidemia and Methylmalonic Acidemia
Jul 15, 2020| Posted in: Congenital And Genetic Conditions, Diagnosis, Drug Development, Neurology/Nervous System Diseases
Ashish Sagrolikar, chief commercial officer of Zogenix, discusses…
Dravet Syndrome Explained: “A Constant State of High Alert”
Jul 8, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Hematologic Disorders, Metabolic Disorders, News, Treatment
Federico Grossi, MD, chief medical officer at Apellis…
Data from Phase 3 Trial Shows Promise for Improved Treatment of PNH
