The Myelin Project

2008 Got the Nerve Duathlon

Fri, 07 Mar 2008 20:47:13 GMT

2008 Got the Nerve Duathlon
Start Date: 11-Oct-08 8:30 AM
End Time: 11-Oct-08 12:00 PM
Location: Palo Curo Canyon State Park, Canyon, Texas
Speaker:

Palo Curo Canyon State Park

2008 Got the Nerve Triathlon

Sat, 02 Feb 2008 19:40:16 GMT

2008 Got the Nerve Triathlon
Start Date: 24-May-08 8:15 AM
End Time: 24-May-08 5:30 PM
Location: Mt. Gretna, Pennsylvania, Mt. Gretna, PA
Speaker: Chris Kaag

Event Details:

The annual Got the Nerve Triathlon will be held for the 2008 season in May. Join us and see more information at this link: 2008 Got the Nerve

Mt. Gretna, Pennsylvania

One Step Closer - Walk for Leukodystrophy

Thu, 01 May 2008 15:48:01 GMT

One Step Closer - Walk for Leukodystrophy
Start Date: 17-May-08 12:00 PM
End Time: 17-May-08 4:00 PM
Location: Old Bridge High School - Lombardi Field, Matawan, NJ 07747
Speaker:

Old Bridge High School - Lombardi Field

4209 Rout 516

Matawan, NJ 07747

BAREFOOT TRUTH

Fri, 09 May 2008 14:59:32 GMT

BAREFOOT TRUTH
Start Date: 10-May-08 7:00 PM
End Time: 10-May-08 9:00 PM
Location: Northwood School, Lake Placid, New York 12948
Speaker: Baretooft Truth

Northwood School

92 Northwood Road

Lake Placid, New York 12948

[firstname] [lastname], The Myelin Project Newsletter 8-May-08

noemail@myelin.org — Thu, 08 May 2008 22:21:26 GMT

Untitled Document Hello [firstname], Welcome to the The Myelin Project newsletter of upcoming events, articles, jobs and more from our membership. Articles for 8-Apr-08 to 8-May-08 Outreach Letter to ALD/AMN Families Author: Candace Root Release Date: Thu 8-May-08 5:00 PM Dear ALD/AMN Families: I am writing to let you know about a study of the treatment of adrenomyeloneuropathy (AMN) which is now in progress at the Kennedy Krieger Institute and Johns Hopkins Hospital, both located in Baltimore, Maryland. Adult males and females with AMN who experience symptoms but who are, at minimum, able to walk 20 yards with crutches, canes, or a brace are eligible to enroll. The primary purpose of the study is to determine whether Lorenzo’s Oil (a 4:1 mixture of glyceryl... [More Info] Posted by: Candace Root GOOD...

8-May-08 5:21 PM

NEWBORN SCREENING TEST

noemail@myelin.org — Thu, 08 May 2008 22:00:00 GMT

Dr. Gerald Raymond Reports on Newborn Screening for X-linked Adrenoleukodystrophy and Other Peroxisomal Disorders X-linked adrenoleukodystrophy (ALD) is a genetic disorder occurring in 1 in 17,000 newborns. All ethnic groups are affected about equally. The disease causes a progressive loss of brain white matter and loss of adrenal gland function in boys who have the gene. It strikes previously healthy boys beginning school or young men just starting out in life. Over a third of boys with the biochemical abnormality will show symptoms in childhood. Without treatment, they will be severely disabled within a few months and will die in childhood. There are effective therapies for ALD, including replacement of adrenal hormones, bone marrow therapy for early brain disease, and the investigational use of Lorenzo’s oil. These therapies work best if started before symptoms develop. Until now, diagnosis has relied on the identification of an affected individual and then the systematic...

8-May-08 5:00 PM

Pelizaeus-Merzbacher Disease

noemail@myelin.org — Thu, 08 May 2008 22:00:00 GMT

Pelizaeus-Merzbacher Disease Application of a new progesterone antagonist to reduce PLP overexpression in a mouse model of Pelizaeus-Merzbacher Disease by Klaus-Armin Nave, PhD, Hauke Werner, PhD Max-Planck Institute of Experimental Medicine, Goettingen, Germany Pelizaeus-Merzbacher disease (PMD) is a severe leukodystrophy defined by mutations of the X chromosome-linked gene for proteolipid protein (PLP), the major myelin protein of the central nervous system (CNS). The majority of patients suffer from a PLP-gene duplication and, in consequence, PLP overexpression that is not tolerated by myelin-producing oligodendrocytes. We are testing the hypothesis that PLP overexpression can be pharmacologically lowered to improve the disease course. Although a detailed knowledge of PLP gene regulation in vivo is still lacking, there is indirect evidence to suggest that the steroid hormone progesterone stimulates the PLP gene in oligodendrocytes, similar to the PMP22 gene in...

8-May-08 5:00 PM

GOOD NEWS FOR MLD PATIENTS

noemail@myelin.org — Thu, 08 May 2008 22:00:00 GMT

Good news for MLD Patients There is some very good news today for metachromatic leukodystrophy (MLD) patients and their families. The U.S.. Food and Drug Administration (FDA) has granted Orphan Drug status to Zymenex for Metazym, and approved the start of Phase II Clinical Trials. This step forward is particularly good news for The Myein Project. Through the British Trust for The Myelin Project, our branch in the United Kingdom, The Myelin Project has helped to fund development of this MLD treatment. Metazym is a preparation of arylsulfatase A, the enzyme that is not working correctly in MLD. By giving patients the enzyme, it is hoped that further destruction of myelin will be stopped. I would like to extend my sincere thanks to our generous donors whose contributions helped make this step possible. What is an Orphan Drug? An orphan drug is one that is developed for the treatment of a rare disease; that is, a disease affecting fewer than 200,000 persons. The U.S. Congress...

8-May-08 5:00 PM

Outreach Letter to ALD/AMN Families

noemail@myelin.org — Thu, 08 May 2008 22:00:00 GMT

Dear ALD/AMN Families: I am writing to let you know about a study of the treatment of adrenomyeloneuropathy (AMN) which is now in progress at the Kennedy Krieger Institute and Johns Hopkins Hospital, both located in Baltimore, Maryland. Adult males and females with AMN who experience symptoms but who are, at minimum, able to walk 20 yards with crutches, canes, or a brace are eligible to enroll. The primary purpose of the study is to determine whether Lorenzo’s Oil (a 4:1 mixture of glyceryl trioleate and glyceryl trierucate) helps patients with AMN. This part of the study is placebo-controlled, that is, half of the patients receive Lorenzos Oil, while the other half receives a placebo oil. The other part of the study is an individualized physical therapy program, customized to address each patient’s needs based upon a detailed analysis of their muscle strength, degree of spasticity, sensation, balance and gait. This comprehensive analysis is conducted using state-of-the-art...

8-May-08 5:00 PM

A REVIEW OF RECENT PROGRESS

noemail@myelin.org — Wed, 07 May 2008 16:00:00 GMT

The Myelin Project was established in 1989 with the aim of funding research on the demyelinating diseases, such as the leukodystrophies and multiple sclerosis. The organization was founded by Augusto Odone and his late wife, Michaela. Their son, Lorenzo, suffers from X-linked adrenoleukodystrophy (X-ALD), the most common of the leukodystrophies. Research has been focused on efforts to understand mechanisms of myelin repair, with a view to discovering therapies that will permit restoration of damaged myelin. It is anticipated that these will permit at least partial restoration of function in patients suffering from myelin loss. Mr. Odone served as president of The Myelin Project from its inception until January of this year, when poor health forced his retirement. At that time, the Myelin Project Board of Directors voted to name Margaret T. Weis, Ph.D., an Associate Professor in the School Of Pharmacy at Texas Tech University Health Sciences Center in Amarillo, as president. The U.S....

7-May-08 11:00 AM

The Myelin Project Newsletter 19-Dec-07

noemail@myelin.org — Wed, 19 Dec 2007 22:00:00 GMT

THE MYELIN PROJECT - Winter Newsletter 2007 THE STATUS OF REMYELINATION We can do better. With your continued support, we will.’ by Dr. Margaret Weis Although I do not have an immediate family member affected by a demyelinating disease, I do have first-hand knowledge of the daily struggles to take care of a beloved child suffering from one of these devastating disorders. To each affected family, the wait for newer and more effective therapies must surely seem endless. Even to me, a practicing scientist, the pace of research (especially my own) often appears to...

19-Dec-07 4:00 PM

Adrenoleukodystrophy: incidence, new mutation rate, and results of extended family screening.

noemail@myelin.org — Fri, 30 Nov 2007 20:00:00 GMT

Below is the abstract for full text of this article visit Pub Med : Ann Neurol. 2001 Apr;49(4):512-7

Utilizing the plasma very long chain fatty acid assay, supplemented by mutation analysis and immunofluorescence assay, we determined the number of X-linked adrenoleukodystrophy (X-ALD) hemizygotes from the United States identified each year in the two laboratories that perform most of the assays in this country: the Kennedy Krieger Institute between 1981 and 1998 and the Mayo Clinic Rochester from 1996 to 1998. The minimum frequency of hemizygotes identified in the United States is estimated to be 1:42,000 and that of hemizygotes plus heterozygotes 1:16,800. Our studies involved 616 pedigrees with a total of 12,787 identified at-risk members. Diagnostic assays were performed in 4,169 at-risk persons (33%) and included members of the extended family. Only 5% of male probands and 1.7% of X-ALD hemizygotes were found to have new mutations. The extended family testing led to the identification of 594 hemizygotes and 1,270 heterozygotes. Two hundred fifty of the newly identified hemizygotes were asymptomatic and represent the group in which therapy has the greatest chance of success. Identification of heterozygotes provides the opportunity for disease prevention through genetic counseling. Diagnostic tests should be offered to all at-risk relatives of X-ALD patients and should include members of the extended family.

PMID: 11310629 [PubMed - indexed for MEDLINE]

30-Nov-07 2:00 PM

Therapy for Lorenzo's Oil Boys

noemail@myelin.org — Tue, 30 Oct 2007 23:00:00 GMT

Therapy for Lorenzo's Oil Boys

30-Oct-07 5:00 PM

KYLE'S STORY

noemail@myelin.org — Sat, 20 Oct 2007 22:00:00 GMT

A Divine Appointment: Kyle’s Story This is a simple account of events which transpired on July 16, 2007 at a very different company called I Am Unique Special Care and Case Management, Inc., a home care agency. The company exists because of the direction of the Lord, I Am. Every individual is Unique and Special Care is given to each client. The Case Management is delivered with a standard of excellence, integrity, values, and moral ethics, often going far beyond what is routinely expected in home care. The Glass family is a part of IAU. There’s Mom Lisa; Dad Rodney, client Kyle; his brother Brett, and I can’t fail to mention Zero the dog whose only claim to fame is that he’s loved by this family. There are several nurses who provide Kyle’s care, but his lead nurse is, Cynda Blakely. She loves the Lord and believes in the power of a prayer. Kyle has one of those rare parent’s worst nightmare diseases, Adrenoleukodystrophy or ALD. It is a ravaging neuro-degenerative disease. Symptoms...

20-Oct-07 5:00 PM

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...

MESSAGE FROM THE EXECUTIVE DIRECTOR

Fri, 09 May 2008 15:18:01 GMT

Dear Myelin Project Supporters: Thank you for the tremendous response to our new web site. We hope to be able to serve you better and more efficiently with our new web site and welcome your comments and input. We are currently developing a medical/science section of our web site to be used as a reference tool for health care professionals. I would like to take this opportuntity to welcome our new Development Director, Trish Knight. Trish is the founder and President of the Stennis Foundation, a non-profit foundation that raises money to fund leukodsytrophy research. Trish will be developing national fund raising and will also be working with our leukodsytrophy families. If you have a family member with leukodystrophy please contact her at trish.knight@myelin.org She would love to hear from you. She is also compiling a data base of leukodsytrophy patients so please contact her so that she can include you in the data base. If you want to host a fund raiser for The...

Fri, 09 May 2008 15:10:13 GMT

For an update on recent scientific and medical news please see Articles.

Photos of recent events, and find out what's happening in the Myelin Project world.

Photographs of the Hammerfest Triathlon were taken by Kate Berges and were used with permission www.kateberges.com

Photographs of the 12 in 24 Jump for a Cause were taken by Michelle Dobson, our fabulous lone female sky diver.

LETTER FROM THE PRESIDENT

Thu, 08 May 2008 21:42:11 GMT

March 6, 2008 Dear Friends of The Myelin Project: 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...

Outreach Letter to ALD/ALM Families

Thu, 08 May 2008 21:22:28 GMT

GOOD NEWS FOR MLD PATIENTS

Thu, 08 May 2008 21:16:26 GMT

There is some very good news today for metachromatic leukodystrophy (MLD) patients and their families. The U.S.. Food and Drug Administration (FDA) has granted Orphan Drug status to Zymenex for Metazym, and approved the start of Phase II Clinical Trials. This step forward is particularly good news for The Myein Project. Through the British Trust for The Myelin Project, our branch in the United Kingdom, The Myelin Project has helped to fund development of this MLD treatment. Metazym is a preparation of arylsulfatase A, the enzyme that is not working correctly in MLD. By giving patients the enzyme, it is hoped that further destruction of myelin will be stopped. I would like to extend my sincere thanks to our generous donors whose contributions helped make this step possible. What is an Orphan Drug? An orphan drug is one that is developed for the treatment of a rare disease; that is, a disease affecting fewer than 200,000 persons. The U.S. Congress passed the Orphan Drug Act in...

DEVELOPMENT

Thu, 08 May 2008 14:08:37 GMT

It’s All About Development

Trish Knight
Director of Development

Development is a new area within The Myelin Project, and this new area is in exactly that stage: the development stage. We are planning our strategies as to how we can further the research of demyelinating diseases, including the Leukodystrophies and Multiple Sclerosis; as

well as bring about the implementation of Newborn Screening for Adrenoleukodystrophy (ALD). For boys who are diagnosed with ALD, treatment with Lorenzo’s Oil and a bone marrow

transplant could slow the progression of the disease. But ultimately, our goal at The Myelin Project is for these diseases to be determined at birth through Universal Newborn Screening. The development of these tests is quite costly; and federal funding is limited. Donations are needed to continue developing these tests, so that children who are diagnosed with ALD or other demyelinating disorders are given a chance at life. It’s all about development.

email Trish.Knight@myelin.org

How Can I Get Lorenzo's Oil?

Wed, 07 May 2008 20:58:19 GMT

In the U.S., Lorenzo's Oil is currently only available to patients taking part in a clinical trial under the direction of Dr. Gerald Raymond of the Kennedy Krieger Institute. He can be reached at (800) 873-3377. Dr. Raymond has assumed the work of the late Dr. Hugo Moser. We are working with the FDA to obtain approval of Lorenzo's Oil as a drug, so that it can be widely available.

In the U.S., Lorenzo's Oil can only be obtained through prescription by Kennedy Krieger authorized physicians. A 500ml bottle costs approximately $56.00. Some insurance companies will provide coverage for the oil, but others do not because it is still considered an experimental drug by the FDA.

The oil is jointly manufactured by Croda International of Britain and SHS International (Scientific Hospital Supply.

If you live outside the United States, please see Lorenzo's Oil International

Scientific Advisory Panel

Wed, 07 May 2008 20:08:59 GMT

Ian Duncan, PhD Chair of Scientific Advisory Panel Professor, Neurology Department of Medical Sciences School of Veterinary Medicine University of Wisconsin-Madison 2015 Linden Drive West Madison, WI 53706-1102 USA Email Duncan@svm.vetmed.wisc.edu Dr. Annik Baron-Van Evercooren Director, Unit on Disorders of Myelin and Muscle Ion Channels Faculté Pitié-Salpêtrière Laboratoire des Pathologies de la Myéline INSERM CJF 97-11 105 boulevard de l’Hôpital 75634 Paris Cedex 13 FRANCE William Blakemore< Ph.D. Professor of Neuropathology University of Cambridge Department of Clinical Veterinary Medicine Madingley Road Cambridge CB3 0ES ENGLAND Wolfgang Bruck, M.D. Professor of Neuropathology Institut fur Neuropathologie Universitatsmedizin Gottingen Robert-Koch-Strabe 40 37075 Gottingen GERMANY Robin J.M. Franklin, PhD Professor of Neuroscience Cambridge Centre for Brain Repair & Centre for Veterinary Sciences ...

Lorenzos Oil International

Wed, 07 May 2008 19:44:16 GMT

Latin America Alex Gooding email ahgooding@sbinshs.com.ar Tel +54 11 493 24816 Far East Okke Suurenbroek email okkesurrenbroek@nutriciachina.net Tel +8621 5351 6858 Middle East/Africa/India Rosenda Nair email NRose@nutricia.com.ae Tel +971 4 8056736 Argentina ABIN S.A. Cochabamba 3641 1252 Buenos Aires Argentina Tel: +54 11 495 76764 Fax: +54 11 481 36697 Australia Nutricia Australia Pty Ltd P.O. Box 1007 North Ryde Business Centre NSW 1670 Talavera Corporate Centre Level 4, Building D 12-18 Talavera Rd Macquarie Park NSW 2113 Tel: +61 2 8870 0400 Fax: +61 2 9878 4725 Austria Nutricia Nahrungsmittel GmbH & Co.Kg Jochen-Rindt-Strasse 37 A-1230 Vienna Tel: +43 - 1 - 6882626 Fax: +43 - 1 - 6882626 Belgium N.V. Nutricia België Rijksweg 64 B-2880 Bornem Tel: +32 - 3 - 890 22 11 Fax: +32 - 3 - 890 22 12 Brazil Support Products Nutricionais Ltda. Rua Gomes de Carvalho, 1510 - 18° Andar Vila Olimpia São, Paulo, SP Tel: 55 (11)...

Pelizaeus-Merzbacher Disease

Wed, 07 May 2008 16:59:19 GMT

Application of a new progesterone antagonist to reduce PLP overexpression in a mouse model of Pelizaeus-Merzbacher Disease by Klaus-Armin Nave, PhD, Hauke Werner, PhD Max-Planck Institute of Experimental Medicine, Goettingen, Germany Pelizaeus-Merzbacher disease (PMD) is a severe leukodystrophy defined by mutations of the X chromosome-linked gene for proteolipid protein (PLP), the major myelin protein of the central nervous system (CNS). The majority of patients suffer from a PLP-gene duplication and, in consequence, PLP overexpression that is not tolerated by myelin-producing oligodendrocytes. We are testing the hypothesis that PLP overexpression can be pharmacologically lowered to improve the disease course. Although a detailed knowledge of PLP gene regulation in vivo is still lacking, there is indirect evidence to suggest that the steroid hormone progesterone stimulates the PLP gene in oligodendrocytes, similar to the PMP22 gene in Schwann cells (Sereda et al, 2003)....

Lorem ipsum survey

noemail@myelin.org — Thu, 09 Aug 2007 20:40:38 GMT

Objectives:

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Duis autem dolor in hendrerit in vulputate velit esse molestie consequat, vel illum dolore eu feugiat nulla facilisis at vero eros et accumsan et iusto odio dignissim qui blandit praesent luptatum zzril delenit au gue duis dolore te feugat nulla facilisi.

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File uploaded by Candace Root.

Lorem ipsum

2007-08-09T20:40:37Z

Instructor: Instructor

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