Rare Diseases Activities
The Challenges in Long-Chain Fatty Acid Oxidation Disorders: Unmet Needs of Treatment and Management
This supplement highlights the pathophysiology and clinical findings for this rare group of genetic disorders, management strategies including and clinical trial data for the recently approved therapy, as well as safety and efficacy clinical trial data of agents under investigation.
Opportunities for Delivering Optimal Care in Spinal Muscular Atrophy
This activity will examine the epidemiology, pathophysiology, clinical presentation, and diagnosis of spinal muscular atrophy (SMA).
Treating Chronic Inflammatory Demyelinating Polyneuropathy With Immunoglobulin: Strategies for Appropriate Use and Accurate Dosing
This activity will examine the pathophysiology of chronic inflammatory demyelinating polyneuropathy (CIDP).
Current Treatment Options for Atypical Hemolytic Uremic Syndrome: The Role of the Specialty and Infusion Pharmacist
This activity will feature videos including demonstration of the preparation of these biologic agents as well as patient counseling vignettes to enhance pharmacists’ knowledge and ability to make interventions to optimize the care of patients with aHUS.