Metabolic Disorders
Oct 20, 2020| Posted in: Cancers, Diagnosis, Endocrine Disorders, Metabolic Disorders, Skin Conditions
Susan Samson, MD, PhD, of Baylor College of…
Diagnostic Delays are Common for Acromegaly
Oct 19, 2020| Posted in: Cancers, Endocrine Disorders, Metabolic Disorders, Skin Conditions
Lisa Nachtigall MD, Clinical Director, Neuroendocrine and Pituitary…
Acromegaly Overview
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 14, 2020| Posted in: Cancers, Endocrine Disorders, Metabolic Disorders, Treatment
Susan Samson, MD, PhD, of Baylor College of…
Treatment Options for Acromegaly
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
Sep 27, 2020| Posted in: Advocacy, Congenital And Genetic Conditions, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases
Kelly Heger, Founder of the AADC Family Network, provides…
Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Overview
Sep 22, 2020| Posted in: Congenital And Genetic Conditions, Gastrointestinal Diseases, Metabolic Disorders, Treatment
Kealey-Shay Spolijarevic has progressive familial intrahepatic cholestasis (PFIC),…
PFIC Patient Describes Treatment Options
Sep 17, 2020| Posted in: Congenital And Genetic Conditions, Metabolic Disorders, Neurology/Nervous System Diseases, News, Ophthalmology/Eye Diseases, Regulations
The U.S. Food and Drug Administration (FDA) has…
FDA Fast-tracks Review Process for Drug to Treat Niemann-Pick Type C
Sep 16, 2020| Posted in: Congenital And Genetic Conditions, Expert Perspectives, Heart Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases
In this 4 module CME/CE program, hosted by…
New CME/CE focused on Mucopolysaccharidosis I (MPS I)
Sep 15, 2020| Posted in: Advocacy, Gastrointestinal Diseases, Metabolic Disorders
Kealey-Shay Spolijarevic gives a message to other PFIC…
Your Life Will be Different – A Message to PFIC Patients
