Drug Development
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 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 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: 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 11, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Congenital And Genetic Conditions, Drug Development, Metabolic Disorders, News
Podcast: Clinical Data Shows Promise for SCID Treatment
Judy Shizuru, MD, Blood and Marrow Transplant Specialist,… Jan 11, 2021| Posted in: Drug Development, Endocrine Disorders
Hypothalamic Obesity Study with Tesomet Showing Promise
Ulla Feldt-Rasmussen, MD, Department of Medical Endocrinology and… Jan 7, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Neurology/Nervous System Diseases
PROOF-HD: Pivotal Huntington Disease Trial Underway
Michael Hayden, PhD, Chief Executive Officer of Prilenia Therapeutics,… 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
Orphan Drugs Currently Under Review by the FDA
Every potential Food and Drug Administration (FDA) approval… Dec 24, 2020| Posted in: Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Drug Development, Hematologic Disorders