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NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced the company has initiated enrollment of PARADIGHM, a global natural history registry for patients with hypoparathyroidism. Patients will be enrolled and followed for at least 10 years to characterize this rare, complex endocrine disorder under conditions of normal clinical practice.
Hypoparathyroidism is a rare endocrine disorder in which the body produces insufficient levels of parathyroid hormone, the principal regulator of calcium and phosphorus. When the body has too little parathyroid hormone, blood calcium levels drop and phosphorus levels increase, which can cause a multitude of nearly 40 physical, emotional and cognitive symptoms, including uncontrollable muscle spasms and cramps, tetany, seizures, fatigue, anxiety and depression.
“A common challenge in the rare disease space is that the disorders are often misunderstood, due to a lack of fundamental information characterizing the complications and long-term risks,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharmaceuticals. “We are launching PARADIGHM to create a comprehensive database that builds our understanding of the nature of hypoparathyroidism and its burden, so that the unmet needs of patients may ultimately be better addressed.”
There is limited data illustrating the fundamental aspects of hypoparathyroidism. The objective of PARADIGHM is to fill this scientific need by characterizing the natural history of chronic hypoparathyroidism, including its treatment, clinical outcomes, comorbidities and mortality as observed in a typical clinical setting. The ultimate goal for compiling the PARADIGHM registry is to assist healthcare practitioners in optimizing clinical decision making for patients with hypoparathyroidism. The registry is designed to help healthcare practitioners better understand the variability, progression and natural history of the disorder.
Physicians Advancing Disease Knowledge in Hypoparathyroidism (PARADIGHM) is a prospective, observational, natural history registry that is open to patients with chronic hypoparathyroidism, regardless of age, gender, etiology or management of the disorder. No study-defined procedures will be required and a select data set will be collected at baseline and at least annually, as available.