Gastrointestinal Diseases

Disease overviews, expert insight, and general news into relevant research on rare gastrointestinal diseases.

Complement-Mediated Kidney Disorders: A Case Series

This CME-accredited program, developed by Howard Trachtman and Carla M. Nester, utilizes three unique case studies to address clinical questions which explore the complexities involved in diagnosing, treating, and managing patients with various complement-mediated kidney disorders.

FcRn and Myasthenia Gravis: Treatment Options

Richard J. Nowak, MD, MS, discusses the safety and efficacy of neonatal fragment crystallizable receptor (FcRn)-directed therapies for patient with myasthenia gravis.

Epigenetic Modifiers as Therapeutic Targets

Gaucher disease (GD) is a genetic disorder in which glucocerebroside accumulates in cells and certain organs. The disorder is characterized by bruising, fatigue, anemia, low blood platelet count and enlargement of the liver and spleen, and is caused by a hereditary deficiency of the enzyme glucocerebrosidase, which acts on glucocerebroside.

Optimizing the Efficacy and Safety of Therapy for Fabry Disease

Fabry disease is an inherited disorder that results from the buildup of a particular type of fat in the body’s cells, called globotriaosylceramide or GL-3. The disorder affects many parts of the body.

FcRn and Myasthenia Gravis

This half-hour CME-accredited program, hosted by Richard J. Nowak, MD, MS, explains the role of neonatal fragment crystallizable receptor (FcRn) in myasthenia gravis (MG) and how treatments that target FcRn are being used to manage patients with MG.

FcRn and Myasthenia Gravis: Pathophysiology

Richard J. Nowak, MD, MS, explains the role of neonatal fragment crystallizable receptor (FcRn) in myasthenia gravis (MG).

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Plans for a Phase 3 Clinical Trial Evaluating FLT201 in Patients With Gaucher Disease