The Myelin Project

Zymenex sells clinical candidate Metazym to Shire

noemail@myelin.org — Thu, 08 May 2008 21:00:00 GMT

PRESS RELEASE April 24th, 2008 Zymenex sells clinical candidate Metazym to Shire for 135 MUSD Zymenex has – with the financial support of BankInvest Biomedical Venture and Sunstone Capital – achieved successful early clinical development of a therapeutic candidate Metazym against the rare, lethal and genetically caused, disease MLD, which most often affects children at a very young age. This has now resulted in a sale of the therapeutic project to the global specialty biopharmaceutical company Shire plc. Shire plc, which is quoted on the London Stock Exchange and Nasdaq, is one of the world’s largest specialty biopharmaceutical companies. Shire expects - after the acquisition of the Metazym project – to continue development and to seek to bring the product to market as quickly as possible. Shire, through its Human Genetic Therapies business, is a leading player in the development and commercialization of treatments against a number of lysosomal storage diseases, of...

Zymenex gets US Orphan Approval

noemail@myelin.org — Fri, 18 Apr 2008 22:00:00 GMT

P R E S S R E L E A S E Hillerød, Denmark, April 15, 2008 Zymenex gets US Orphan Approval The FDA has granted Zymenex Orphan Designation for its enzyme Metazym, for the treatment of the rare, lysosomal disease Metachromatic Leukodystrophy (MLD). Orphan designation qualifies the sponsor of the product for tax credits and marketing exclusivity incentives of the Orphan Drug Act. Zymenex has previously received Orphan Designation in the EU. Zymenex has just recently also received US FDA approval of its Investigational New Drug (IND) application, for its enzyme Metazym. Based on the successfully completed EU Phase 1 trial results, the company can go directly into Phase 2 clinical trials in the US. The US Phase 2 clinical trial is being conducted by Dr. Maria Escolar at the Program for Neurodevelopmental Function in Rare Disorders, Center for the Study of Development and Learning, University of North Carolina (UNC), Chapel Hill, North Carolina, USA. Metazym is in Phase 2...

DEVELOPMENT OF NEWBORN SCREENING TEST FOR ADRENOLEUKODYSTROPHY

noemail@myelin.org — Fri, 07 Mar 2008 18:00:00 GMT

When the Myelin Project was founded by Augusto and Michaela Odone, the little was known about ALD. Now, twenty years later, we not only know a great deal about the disease, we have treatments that, however imperfect, offer hope of survival for boys who carry the ALD gene. As published in 2005, Lorenzo’s Oil significantly reduces the risk of childhood cerebral ALD in boys carrying the ALD gene. Studies are now underway to discover whether Lorenzo’s oil will delay the onset and progression of adrenomyeloneuropathy in men and women who carry the ALD gene. There is also hope for those boys who carry the gene and develop symptoms of childhood cerebral ALD. When bone marrow transplant was first tried, the results were often very disappointing. Today, though, with advances in matching donors and recipient, and better pre- and post-operative therapies, the success rate is very high. The good news is that these advances give us hope that the specter of childhood cerebral ALD will be...

New Developments

noemail@myelin.org — Wed, 31 Oct 2007 20:00:00 GMT

http://news.bbc.co.uk/2/hi/health/7067899.stm

Early Detection Called Key to Effectiveness

noemail@myelin.org — Sat, 01 Jan 2005 21:00:00 GMT

By Carey Goldberg, Globe Staff | July 12, 2005 New research offers scientific vindication for the parents whose fight to save their ailing son inspired the 1992 movie Lorenzos Oil. It comes too late to help the real-life Lorenzo, who is nearly totally paralyzed, but suggests that the oil could help save future generations of boys from the same, fatal nerve disorder. The paper, published yesterday in the Archives of Neurology, found that the oil was often effective at fending off symptoms in young boys who had the genetic disease that has devastated Lorenzo Odone. Among the 89 boys who started taking the oil before symptoms appeared, only 11 percent went on to develop them, compared with the usual 35 percent. In a prototypical clash between activist parents and resistant doctors, the medical establishment has been extremely skeptical about the effectiveness of Lorenzos oil. Among those skeptics was Dr. Hugo W. Moser, a professor at Johns Hopkins University and the lead author of the...