Congenital and Genetic Conditions
Oct 21, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Treatment
Jim Willson, a patient with spinal muscular atrophy…
SMA Patient Discusses His Experience with Risdiplam
Oct 19, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Drug Development, Treatment
Mary Smith, PhD, senior vice president of clinical…
Nirogacestat’s Mechanism of Action to Treat Desmoid Tumors
Oct 17, 2020| Posted in: Advocacy, Congenital And Genetic Conditions, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, News
Joslyn Crowe, executive director of the National Niemann-Pick…
How Families Cope with a New Rare Disease Diagnosis
Oct 15, 2020| Posted in: Congenital And Genetic Conditions, Endocrine Disorders, Expert Perspectives, Kidney And Urinary Diseases, Musculoskeletal Diseases, Treatment
Erik Harris, Executive Vice President and Chief Commercial…
Approval of Burosumab-twza for Treating Tumor-Induced Osteomalacia
Oct 14, 2020| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases
Jim Willson, a patient with spinal muscular atrophy…
SMA Adult Talks About His Diagnostic Journey
Oct 12, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Treatment
Mary Smith, PhD of SpringWorks Therapeutics explains the…
Past, Present, and Future Treatment Options for Desmoid Tumors
Oct 8, 2020| Posted in: Congenital And Genetic Conditions, Heart Diseases, Metabolic Disorders, Neurology/Nervous System Diseases
Erik Harris, Executive Vice President and Chief Commercial…
Triheptanoin Approved to Treat Long-chain Fatty Acid Oxidation Disorders
Oct 7, 2020| Posted in: Cancers, Congenital And Genetic Conditions
Mary Smith, Ph.D. of SpringWorks Therapeutics explains the…
What are Desmoid Tumors
Oct 5, 2020| Posted in: Advocacy, Congenital And Genetic Conditions, Heart Diseases, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases
Tiffany Cook, Family Resource Manager of CureDuchenne, discusses…
Video Game Tournament Provides Support for Duchenne Patients
Oct 1, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Endocrine Disorders, Neurology/Nervous System Diseases, Treatment
Jörn-Sven Kühl, MD, Department of Pediatric Oncology, Hematology…
Gene Therapy Shows Promise for Cerebral Adrenoleukodystrophy (CALD)
