LEHI -- Kaleb Heaps was a tenacious 9-year-old who enjoyed playing tackle football, riding dirt bikes and snowboarding. Now he's a tenacious 9-year-old who enjoys all those things in spite of a diagnosis five months ago of medullablastoma, a form of brain cancer. Since then, this bright-eyed boy has endured surgery, 60 rounds of radiation and several rounds of chemotherapy. Described as loving, witty, courageous and charming, Kaleb seems to have an unbreakable spirit that has helped him keep up with the demands of his illness. "Kaleb is the toughest kid I know," his dad, Brian Heaps, said. "He has the best attitude. I think that a lot of people would be pretty upset. He is a special boy. He has a big heart." The treatment has caused ataxia and the left side of his body does not work correctly. Kaleb is unable to walk without a walker or other assistance. He has double vision and wears an eye patch to help correct the problem. Kaleb has accepted these challenges in stride, dressing as a pirate last Halloween while at Primary Children's Medical Center. "It is a good time to get an eye patch before you do Halloween," Kaleb said … Continue reading →

To: HEALTH, MEDICAL AND STATE EDITORS SPRINGFIELD, Va., March 15, 2012 /PRNewswire-USNewswire/ -- Today, Repligen Corporation announced enrollment of the first patient in a Phase 1 clinical trial of RG2833 in adult patients with FA -- a debilitating, life-shortening, degenerative neuro-muscular disorder that affects children and adults and currently has no treatment. RG2833 is a class 1 Histone Deacetylase (HDAC) inhibitor specifically designed to increase frataxin protein production in FA patients. This therapeutic approach targets the genetic mutation that is the fundamental cause of the disease. (Logo: http://photos.prnewswire.com/prnh/20111226/DC27366LOGO-a) The potential for these HDAC inhibitors as a therapy for FA was first demonstrated by Dr. Joel Gottesfeld and his colleagues at The Scripps Research Institute in La Jolla, CA. Repligen has worked with Dr. Gottesfeld and other FA scientists around the world to optimize and develop these HDAC inhibitors and prepare them for human clinical trials. FARA is proud to have funded the Gottesfeld's lab's earlier work in which he showed that class 1 HDAC inhibitors could address the basic cause of FA by increasing transcription of the FA gene and production of frataxin protein in cells from FA patients and in FA animal models. "All of us in the global … Continue reading →

(RTTNews.com) - Repligen Corp. (RGEN) said Thursday it has enrolled its first patient in a Phase 1 clinical trial of RG2833 in adult patients with Friedreich's ataxia, or FA. FA is an inherited neurodegenerative disease caused by low levels of the protein frataxin which results in symptoms that typically present in childhood and lead to progressive loss of muscle and nerve function, often resulting in loss of life by early adulthood. RG2833 is an orally bioavailable, class 1 histone deacetylase inhibitor (HDACi) specifically designed to increase frataxin production in patients with FA. The study is being conducted in Turin, Italy and is the first clinical trial of a drug that targets the core genetic defect in FA. The Phase 1 trial is a single ascending dose, crossover study in up to 20 adult FA patients. It is designed to evaluate the pharmacokinetic and safety profile of RG2833. Repligen has previously received U.S. Orphan Drug and European Orphan Medicinal Product designations for RG2833 for the treatment of FA. For comments and feedback: contact editorial@rttnews.com http://www.rttnews.com Originally posted here: Repligen Enrolls First Patient In Study Of RG2833 To Treat Friedreich's Ataxia … Continue reading →

WALTHAM, Mass.--(BUSINESS WIRE)-- Repligen Corporation (NASDAQ:RGEN - News) announced today that it has enrolled its first patient in a Phase 1 clinical trial of RG2833 in adult patients with Friedreichs ataxia (FA). FA is an inherited neurodegenerative disease caused by low levels of the protein frataxin which results in symptoms that typically present in childhood and lead to progressive loss of muscle and nerve function, often resulting in loss of life by early adulthood. RG2833 is an orally bioavailable, class 1 histone deacetylase inhibitor (HDACi) specifically designed to increase frataxin production in patients with FA. This study is being conducted in Turin, Italy and is the first clinical trial of a drug that targets the core genetic defect in FA. The Phase 1 trial is a single ascending dose, crossover study in up to 20 adult FA patients. It is designed to evaluate the pharmacokinetic and safety profile of RG2833. Importantly, this study will also evaluate the pharmacodynamic response of RG2833 on various cellular and molecular biomarkers, including frataxin mRNA and frataxin protein. This Phase 1 trial in patients will generate valuable information on the safety and pharmacology of RG2833, said Walter C. Herlihy, Ph.D., President and Chief Executive Officer … Continue reading →