Musculoskeletal Diseases
Apr 14, 2021| Posted in: Congenital And Genetic Conditions, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases
The Diagnostic Journey For MPS VII Patients
Deborah Marsden, MD, Global Medical Expert, Medical Affairs,… Apr 13, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Musculoskeletal Diseases
FSHD Patient Encourages Others to Get Involved with Patient Advocacy Groups
Amy Bekier, facioscapulohumeral muscular dystrophy (FSHD) patient and… Apr 13, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Musculoskeletal Diseases
Advice for New SMA Parents
Stacy Pokorny, a mother of a young girl… Apr 9, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Metabolic Disorders, Musculoskeletal Diseases, Ophthalmology/Eye Diseases
What is Sly Syndrome
Deborah Marsden, MD, Global Medical Affairs Lead at… Apr 6, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Musculoskeletal Diseases
FSHD Patient’s Diagnostic Journey
Amy Bekier, facioscapulohumeral muscular dystrophy (FSHD) patient and… Apr 1, 2021| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases
Achondroplasia and Bone Lengthening Surgery
Chandler Crews, a young adult with achondroplasia, explains… Apr 1, 2021| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases
Advice for New SMA Parents
Stacy Pokorny, a mother of a young girl… Mar 31, 2021| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases
The Typical Diagnostic Journey for FSHD Patients
Sarah Boyce, President and CEO of Avidity Biosciences,… Mar 30, 2021| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases
“I Call Myself Gadget Girl”: How One Patient Manages Her FSHD
Amy Bekier, facioscapulohumeral muscular dystrophy (FSHD) patient and… Mar 26, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Gastrointestinal Diseases, Heart Diseases, Kidney And Urinary Diseases, Metabolic Disorders, Musculoskeletal Diseases, Ophthalmology/Eye Diseases, Treatment