We are advancing our proprietary genetic medicines platform as we seek to transform the lives of patients with rare diseases by targeting or addressing the underlying cause of the disease. The person, family members and caregivers affected by rare disease are the biggest drivers in every decision our Company makes. From hiring new employees to determining new research initiatives to beginning development programs, we consider the patient community first and foremost.
We continuously seek opportunities to learn from the patients we serve and to provide opportunities to share information about our programs. While we are still in the early stages of clinical development, we are moving forward as quickly as possible, knowing that there are people waiting on new advances. You can learn more about our therapeutic areas or email your questions to email@example.com.
Patient Advocacy Resources for PKU
PKU is a rare inborn error of metabolism caused by a mutation in the PAH gene. If left untreated, PKU can result in progressive and severe neurological impairment. Currently, there are no treatment options for PKU that target the underlying genetic cause of the disease. According to the National PKU Alliance, PKU affects nearly 16,500 people in the U.S. with approximately 350 newborns diagnosed each year. The worldwide prevalence of PKU is estimated to be 50,000 people.
Patient Advocacy Resources for MLD
MLD is a rare lysosomal storage disorder caused by a mutation in the ARSA gene leading to progressive and serious neurological deterioration. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within 5-10 years after onset.
Patient Advocacy Resources for Hunter syndrome
MPS II, or Hunter syndrome, is a lysosomal storage disorder leading to toxic lysosomal accumulation of glycosaminoglycans (GAGs). Hunter syndrome occurs in 1 in 100,000 to 1 in 170,000 males. Severe Hunter syndrome causes progressive debilitation and intellectual decline. The severe form leads to life expectancy of 10 to 20 years. There are no treatments currently available to address both cognitive and peripheral organ manifestations.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a rare, acquired life-threatening blood disease caused by mutations in the PIGA gene that results in intravascular hemolysis, or red blood cell destruction, mediated by uncontrolled activation of the complement system. PNH results in thromboses, recurrent pain, severe anemia, kidney disease and impaired quality of life, among other outcomes.
The Homology team participates in events to raise awareness of rare disease and support patient advocacy organizations.