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Pompe Disease support group. Assisting families with unmet medical costs.

Dedicated to raising awareness of the genetic disorder known as MCAD deficiency and the importance of early detection.

In-depth look at the condition including symptoms, diagnosis and treatment issues. Aimed at the professional level.

Information from the National Health Service on conditions, treatments, local services and healthy living.

Nonprofit organization supporting research and the causes and treatment of IH (pseudotumor cerebri). Information about the condition, patient registry, research ...

Consumer health resource center providing information on causes, symptoms and treatment.

Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years.