Portfolio

Enhance your PNH patient care with VOYDEYA. Manage EVH effectively while maintaining the long-acting disease control of ULTOMIRIS or SOLIRIS. Explore the power of plus.

VOYDEYA is designed to treat EVH in adults while ULTOMIRIS or SOLIRIS continues to provide foundational PNH disease control.

KOSELUGO® (selumetinib) is the first and only FDA-approved therapy proven to shrink NF1 PN in pediatric patients

More Than MG is a resource for those living with or caring for a loved one with myasthania gravis. Access tools and hear from others in the MG community

Learn about hypophosphatasia (HPP), its symptoms, and its effect on both adults and children. Find resources and tools to support your HPP journey.

STRENSIQ® (asfotase alfa) is used to treat perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).

STRENSIQ® (asfotase alfa) is used to treat perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).

Learn more about neuromyelitis optica spectrum disorder, the importance of AQP4-IgG antibodies, relapses, terminal complement pathway, and the burden your patients may face.

Learn about neuromyelitis optica spectrum disorder (NMOSD) causes, diagnosis, symptoms, testing, and treatment.

Learn more about NF1 with PNs (neurofibromatosis type 1 with plexiform neurofibromas).

Learn more about NF1 with PNs (neurofibromatosis type 1 with plexiform neurofibromas).

Learn about KOSELUGO® (selumetinib), a treatment for neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN) in pediatric patients 2 years of age and older.

This website provides information about Hypophosphatasia (HPP). HPP is a rare genetic disorder that affects the way your body uses phosphorus. Phosphorus is important for strong bones and teeth, and for muscle and nerve function. People with HPP have low levels of phosphorus in their blood, which can lead to a variety of problems, including bone pain, fractures, muscle weakness, and problems with breathing and swallowing.

AHUS Source provides services to people with autoimmune hemolytic anemia (AHUS). AHUS is a rare and life-threatening blood disorder that occurs when the body’s immune system attacks its own red blood cells.