Case Studies | Healthcare Providers

BraveCommunity.com is pleased to offer a series of interactive case studies for healthcare providers. These case studies have been created by leading experts in the management of rare genetic disorders, and are based on real cases.

Gaucher disease: Bone Imaging Tutorial

Bone involvement in Gaucher disease can include both trabecular and cortical bone compartments; it can affect bone geometry, bone density, the mineralization of bone, and bone marrow, and cause various complications. Learn about several modalities used to assess bone disease including the use of radiographic methods such as conventional radiography, dual-energy x-ray absorptiometry or DXA and quantitative computed tomography or QCT.

View Bone Imaging Tutorial

Ethan – Hunter syndrome

Learn about Ethan, a 6-year-old boy who was referred to his geneticist from an endocrinologist because of high BMI, short stature and joint contractures. Ethan experiences developmental delay, behavioral problems, and skeletal abnormalities.

Learn more about Ethan and Hunter syndrome

Jim – Hunter syndrome

Jim is a 29-year-old individual with a significant family history of Hunter syndrome. Jim has normal intelligence but suffers from many of the somatic effects of Hunter syndrome.

Learn more about Jim's attenuated case of Hunter syndrome

Luke – Sanfilippo syndrome

Learn about the difficult diagnosis of Sanfilippo syndrome (MPS III). Luke began exhibiting symptoms at 18 months, and after exhibiting increasingly difficult behavor and decreased mental development, his pediatrician referred Luke to a psychologist who felt there was a somatic cause for Luke's behavior and referred the family to a geneticist.

Learn more about Luke and Sanfilippo syndrome

Matthew – Gaucher disease: Therapeutic Goals

Learn about Matthew, a 36-year-old male who was diagnosed with Gaucher disease when a physical exam revealed a decreased platelet count. After being diagnosed, his physician reviewed his symptoms and developed a treatment plan based on the Gaucher Therapeutic Goals Assessment.

Learn more about Matthew and his Therapeutic Goals plan

Michael – Fabry disease

Michael is a 49-year-old male who began to experience symptoms of Fabry disease at age 9. It took 26 years before Michael was correctly diagnosed with Fabry disease.

Learn more about Michael and his road to a correct diagnosis

Peter – Fabry Disease

Peter began to experience episodic pain in his extremities and gastrointestinal symptoms at 7 years of age. After 5 years of misdiagnosis, Peter was finally diagnosed with Fabry disease at age 12.

Learn more about the pediatric presentation of Fabry disease and Peter's struggle

Sarah – Fabry disease

Meet Sarah, a 55-year-old female with Fabry disease. Sarah experienced transient symptoms when she was 40 years old. Physicians did not consider Fabry disease until a male family member was diagnosed.

Learn more about Sarah's complicated diagnosis of Fabry disease

Raymond – Hereditary Angioedema

Raymond is a 54 year old patient who did not experience symptoms of Hereditary Angioedema until later in life. After being admitted to the hospital, he had a longer diagnosis journey than most.

Learn more about Raymonds complicated diagnosis of Hereditary Angioedema

Patricia – Hereditary Angioedema

Patricia is a teenage patient who deals with the psychological and financial effects of Hereditary Angioedema.

Learn more about Patricia and her experiences with Hereditary Angioedema