MEDIA KIT

Acute hepatic porphyria (AHP) refers to a family of rare, genetic diseases characterized by potentially life-threatening attacks and, for some patients, chronic debilitating symptoms that negatively impact daily functioning and quality of life. AHP comprises 4 subtypes, each resulting from a genetic defect leading to deficiency in one of the enzymes of the heme biosynthesis pathway in the liver: acute intermittent porphyria, hereditary coproporphyria, variegate porphyria, and ALAD-deficiency porphyria.

Transthyretin-mediated (ATTR) amyloidosis is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins. The misfolded TTR proteins collect as amyloid deposits throughout the body, including the nerves, heart and digestive system, resulting in progressive organ damage. There are two different types of ATTR amyloidosis – hereditary ATTR (hATTR) amyloidosis, which is caused by an inherited variant, or change, in the TTR gene, and wild-type ATTR (wtATTR) amyloidosis, which occurs without a TTR gene variant.

Primary hyperoxaluria (PH) constitutes a group of rare inherited disorders of the liver characterized by the overproduction of oxalate, an end-product of metabolism. There are 3 types of PH: type 1 (PH1), type 2 (PH2), and type 3 (PH3). PH1 is the most common and severe form, accounting for 70% to 80% of all cases. PH1 affects approximately 4 individuals per million in the United States and Europe, with an estimated 1,300 to 2,100 diagnosed cases. In some regions, such as the Middle East and North Africa, the genetic prevalence of PH1 is higher.

Hypertension, also known as high blood pressure, is a complex multifactorial disease clinically defined by most major guidelines as a systolic blood pressure (SBP) of above 140 mm Hg or a diastolic blood pressure (DBP) greater than 90 mm Hg, though some guidelines have a lower threshold of a SBP above 130 mm Hg or a DBP greater than 80 mm Hg. More than one billion people worldwide live with hypertension, and fewer than 20 percent of those affected have it under control.