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Thalassemia Patients and Friends and thalpal Ā© A. Battaglia 2019

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A message for all  parents who are thals. Keeping your iron load under control is an absolute obligation to your children.
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Author Topic: Lasts Update On HQK-1001  (Read 3069 times)
I wish you a speedy recovery
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Location: Kingdom of Bahrain

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« on: June 10, 2010, 05:13:27 AM »

HQK-1001 is an orally administered short chain fatty acid (SCFA) derivative, which has a unique combination of biological effects - induction of fetal globin and stimulation of red blood cell production - that addresses the underlying pathological mechanisms in sickle cell disease and a related disorder, beta thalassemia. Extensive studies in both the laboratory and relevant animal models carried out in transgenic mice and non-human primates have demonstrated the potential therapeutic effects of this compound in treating these serious, chronic illnesses. HemaQuest has obtained an exclusive worldwide license for a series of patents and patent applications from Boston University for these technologies that give it a strong proprietary position.

HQK-1001 completed short-term and chronic animal toxicology studies, which demonstrated that the drug candidate was safe and well-tolerated at and above doses planned for clinical studies. The compound has excellent oral bioavailability, making it an attractive medication for treating chronic disorders, such as sickle cell disease and beta thalassemia. HemaQuest has completed placebo-controlled Phase 1 clinical trials, where orally administered HQK-1001 was demonstrated to be safe and well-tolerated with no significant adverse events. These clinical studies also demonstrated that the compound had an excellent pharmacokinetic profile and evidence of pharmacodynamic effects as documented by significant increases in early red blood cells, known as reticulocytes. HQK-1001 has recently entered proof of concept clinical trials in both beta thalassemia and sickle cell disease. We expect these trials to be completed by mid-2010, which will provide the basis for moving forward with advanced clinical development including pivotal studies beginning in 2011. In addition, HemaQuest has been granted Orphan Drug Designation for HQK-1001 in both sickle cell disease and beta thalassemia

« Last Edit: June 10, 2010, 05:42:14 AM by baloch » Logged
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