New Zealand Pharmaceuticals Announces Collaboration with the NHGRI to Develop DEX-M74 as a Treatment for Hereditary Inclusion Body Myopathy (HIBM)
New Zealand Pharmaceuticals Ltd (NZP), has entered into a collaboration with the National Human Genome Research Institute (NHGRI), part of the US National Institutes of Health (NIH) located in Bethesda, MD, to take the investigational Hereditary Inclusion Body Myopathy (HIBM) therapeutic, DEX-M74, through pre-clinical development followed by initial clinical trials with HIBM patients lead by NHGRI’s William Gahl MD PhD. Two other NIH groups will contribute to the research, including the NIH Therapeutics for Rare and Neglected Diseases (TRND) program and the NIH Clinical Center. DEX-M74 is intended to reduce or halt the progression of HIBM, a debilitating condition of limb muscle tissue. In order to rapidly bring DEX-M74 to the people suffering from this debilitating disorder NZP exclusively licensed related Intellectual Property from the NHGRI. DEX-M74 is believed to be the leading clinical-stage therapeutic that directly targets HIBM, also known as Distal Myopathy with Rimmed Vacuoles (DMRV), Quadriceps Sparing Myopathy (QSM) and GNE related muscle disease. |
|
|
Prevention of rare genetic disorders
There are hundreds of rare, so-called “orphan” diseases that are listed in this site. Beyond Batten Disease Foundation is working to develop one easy and inexpensive blood test to detect the gene mutations for hundreds of rare genetic diseases. The science is possible today due to tremendous advances in genomic technologies. Since many people are unknown carriers of these diseases the foundation plans to develop the test in order for young couples to get a screening done before they plan a baby. This help to prevent occurrence of these diseases. For more information read http://www.beyondbatten.org/. |
|
|
Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
Hereditary Inclusion Body Myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA), a critical component of many muscle proteins, resulting in a deficiency in SA in the muscles of HIBM patients. The effective replacement of the missing SA substrate is theoretically simple, and, in animal models, replacement with SA showed...
Date First Received: May 20, 2011
Last Updated: May 23, 2011
Verified by: Ultragenyx Pharmaceutical Inc, May 2011
Clinical Trial Phase: Phase 1 | Start Date: July 2011
Overall Status: Not yet recruiting
Estimated Enrollment: 24 |
|
|
