United Leukodystrophy Foundation You Are Not Alone

United Leukodystrophy Foundation

You Are Not Alone

What’s inside... Research..........…….......……2-4 Conference Information.........…....................4 Family Conference Registration Form……..............5 Memorials…………................... 6 Honors/ Sympathies.......…......................7 Paula Brazeal, Ron Brazeal and Hugo Moser, M.D.

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Vol. 31 . No. 2 . Spring 2013

Kirsten Kotlarchik, Operations Manager

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Research Cerebrotendinous Xanthomatosis Symposium July 20, 2012 Summarized by William B. Rizzo, M.D. Chairman, Medical & Scientific Advisory Board and Andrea DeBarber, Ph.D. The ULF hosted an international symposium on Cerebrotendinous Xanthomatosis (CTX) on July 20, 2012. The meeting was sponsored by the Sterol and Isoprenoid Diseases Consortium of the NIH’s Rare Diseases Clinical Research Network. The ULF, together with Manchester Pharmaceuticals, Inc. and Sigma Tau, Inc. generously supported the meeting. Dr. Andrea DeBarber (Oregon Health & Science University, Portland, OR) organized the agenda and the sessions were chaired by Dr. Shailesh Patel (Medical College of Wisconsin, Milwaukee, WI) and Dr. William Rizzo (University of Nebraska Medical Center, Omaha, NE). The meeting concluded with remarks by John Wolf, ULF Board of Directors member and CTX Family Liaison (Aloha, OR). CTX is a rare, later onset leukodystrophy with prominent neurologic symptoms and additional organ involvement. For many years, it carried the distinction of being the only leukodystrophy with a specific and effective therapy. Although other leukodystrophies now have emerging treatments, none are as simple as CTX, which consists of oral supplementation with the bile acid chenodeoxycholic acid (CDCA). Nevertheless, CTX is rarely recognized early when symptoms first develop and the diagnosis is typically delayed for many years. By then, the disease has progressed in many patients to the point where CDCA treatment cannot completely reverse their symptoms. It is therefore critical that physicians consider the diagnosis of CTX in their patients and test for it as soon as possible. Over the past several years the ULF, largely through the work of Paula Brazeal, spearheaded efforts to ensure the continued availability of CDCA for CTX patients in the United States. The CTX Symposium was a tribute to her successful efforts to partner with the FDA and the pharmaceutical industry to guarantee that CTX patients will continue to have the treatment they need. Based on this collaborative effort, CDCA was recently approved by the FDA as an Orphan drug for the treatment of CTX and is now available in the U.S.

This Symposium brought together leading CTX investigators along with CTX families, patient advocates and scientist participants from around the world. The speakers discussed the latest research advances in this disease including its clinical features and complications, genetic causes, biochemical abnormalities and the most up-to-date treatments. Patient advocates were able to rub shoulders with the experts and contribute to the meeting in practical ways. Representatives from Manchester Pharmaceuticals and Sigma-Tau were present. Based on the contributions of all Symposium participants pressing new research directions for CTX emerged. The discussion was lively and constructive. The combined experience with CTX brought by all of the participants to this Symposium was unprecedented. What is CTX?  CTX is a rare leukodystrophy associated with other organ symptoms, including chronic diarrhea starting in childhood, juvenile cataracts, and abnormalities in balance, intellectual disability, peripheral neuropathy, dementia and psychiatric symptoms. The distinctive appearance of xanthomas (fatty tumors) in tendons should alert physicians about CTX.  CTX is caused by mutations in a gene that is necessary for synthesis of bile acids (lipids that are required for fat absorption from the diet). This block in bile acid synthesis results in accumulation of precursor lipids, particularly cholestanol, and causes all symptoms of CTX, including formation of xanthomas in tendons and brain.  CTX is easily diagnosed by measuring bile acid-related metabolites in urine or blood, but the diagnosis is usually delayed until the combination of unusual symptoms appear, especially cataracts and xanthomas.  CTX is the easiest leukodystrophy to treat. Orally administered bile acids (CDCA: brand name Chenodal) will prevent symptoms from developing if started early in the disease, and help improve some symptoms even in adults. It is therefore important to diagnose patients as son as possible so they can be treated.  The early diagnoses of CTX by newborn screening would provide a way to detect all affected infants and start therapy at the time of birth. Professor Antonio Federico (Università degli Studi di Siena, Siena, Italy) gave a clinical overview of CTX. Symptoms usually develop in the second and third decade of life, but may also appear in childhood or even infancy. Some patients present with neonatal cholestatic jaundice, but chronic diarrhea or juvenile cataracts are often the first signs. Osteoporosis frequently develops over time. Neurologic involvement is heralded by the insidious appearance of intellectual impairment, ataxia, psychiatric problems, sensory motor neuropathy, myopathy, extrapyramidal signs, dementia and even a Parkinsonian syndrome in some patients. The distinctive appearance of tendinous xanthomas, particularly involving the Achilles tendon, together with the neurologic disease usually prompts the diagnosis of CTX. Brain MRI demonstrates diffuse white matter disease and MR spectroscopy shows reduced concentrations of NAA and increased lactate. Of 30 patients reviewed by Dr. Federico, cataracts were seen in 23 of

United Leukodystrophy Foundation

Spring 2013

Research

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them, 25 had xanthomas, 14 suffered from diarrhea, 23 showed osteoporosis and 26 had intellectual deficiency. The disease is caused by a genetic defect in bile acid synthesis and is inherited as an autosomal recessive trait. As emphasized by all of the speakers, the symptoms may be prevented by early diagnosis and therapy with CDCA. Dr. Maria-Jesus Sobrido (Fundación Pública Galega de Medicina Xenómica, Santiago de Compostela, Spain) discussed the genetic defects that cause CTX. The disease is caused by mutations in the CYP27A1 gene that results in deficiency of sterol 27hydroxylase, an enzyme necessary for synthesis of bile acids by the liver. This mitochondrial enzyme is important in the conversion of cholesterol to bile acids (cholic acid and CDCA). Most mutations identified in CTX patients are point mutations that cause missense, nonsense and splicing errors. Mutations are often private and unique to a family. Among 20 Spanish patients, she found 38 different alleles that cause the disease. There was no discernible correlation between genotype and phenotype (age of onset, symptoms or severity of disease). She pointed out the need for an up-to-date curated mutation database that would be a resource for genetic diagnosis and evaluation of genotype-phenotype correlations. CTX occurs more frequently in certain geographic regions of the world. Dr. Tzipora Falik-Zaccai (Western Galilee Hospital, Naharia, Israel) reported on population-based genetic CTX screening studies in Israel. She described founder mutations in the Druze isolate and North African Jews derived from Morocco. Among the Druze genetic isolate, which was founded more than 1000 year ago, she screened 2600 people and identified 250 genetic carriers (carrier frequency 1:11). The carrier frequency among North African Jews was 1:50 to 1:70 individuals. She found reluctance by some people to get genetic screening owing to a feeling of stigmatization, apprehension and existing social barriers. Nevertheless, two pregnant women underwent prenatal diagnosis for CTX and were found to carry affected fetuses. The babies were born and treated with CDCA beginning at 1 month of age. They are now 7 years old and free of all symptoms. Dr. Sandra Erickson (University of California, San Francisco, CA) described the Cyp27a1 gene knockout mouse model for CTX. The mice have many of the biochemical features of the human disease including impaired bile acid synthesis, and increased cholestanol and bile alcohol metabolites. In plasma, cholestanol accumulation is seen by 3 months of age, but reverts to normal as the animals age. In contrast, brain cholestanol and cholesterol increase progressively over time but they never reach the levels seen in human disease. Despite these biochemical abnormalities, the knockout mice do not develop xanthomas or the severe neurologic manifestations seen in humans with CTX. Some mice develop premature cataracts and show subtle pathologic changes of adrenal gland hypertrophy. The knockout mice appear to escape the more serious symptoms of CTX because their metabolic pathways for bile acid synthesis differ slightly from humans and they are able to adapt to the genetic defect by inducing alternate pathways that mitigate cholestanol accumulation. The genetic background of the mouse strains may also influence expression of the disease. A better animal model for CTX is required to help understand the human disease and to test new treatment strategies for those patients who do not respond to CDCA. Dr. Kenneth Setchell (Cincinnati Children's Hospital Medical Center, Cincinnati, OH) described methods for biochemical diagnosis of CTX, which is one of 9 disorders of bile acid synthesis. The sterol 27-hydroxylase or CYP27A1 enzyme deficient in CTX is involved in multiple pathways for the synthesis of bile acids. The major (classical) pathway is initiated by hydroxylation of the cholesterol nucleus by cholesterol 7-hydroxylase or CYP7A1. The 7-hydroxycholesterol formed is converted by 3hydroxy-5-C27-steroid dehydrogenase to the bile acid precursor 7-hydroxy-4-cholesten-3-one, which can form multiple metabolites that undergo side chain hydroxylation by CYP27A1 in the pathway to bile acids. Other pathways for bile acid synthesis (including the acidic pathway) are initiated by hydroxylation of the cholesterol side chain by CYP27A1. Dr. Setchell has screened more than 11,000 patients for bile acid pathway disorders by measuring bile acid synthetic metabolites in urine, serum or bile using FAB-mass spectrometry. Using only 50 microliters of urine, negative ions of bile acids and related metabolites are readily detected. Deficiency of CDCA and cholic acid along with elevations in bile-alcohol metabolites are typically seen in CTX. Serum cholestanol is elevated, whereas serum cholesterol is normal. He emphasized the importance of screening all infants with neonatal cholestasis since defects in bile acid synthesis, including CTX, can present in the first 2 months of life. Dr. Andrea DeBarber (Oregon Health & Science University, Portland, OR) described an improved, highly sensitive test for CTX that uses less than a drop of blood. The standard blood test for CTX typically measures cholestanol levels, which are elevated about 5- to 10-fold above normal. Dr. DeBarber treated plasma samples with Girard’s P and other derivatizing reagents to form derivatives of bile acid pathway metabolites that are readily detected using LC-ESI-mass spectrometry. CTX patients were found to accumulate 180-fold more of the bile acid precursor 7-hydroxy-4-cholesten-3-one than controls. This bile acid precursor can be 12-hydroxylated to form 712 -dihydroxy-4-cholesten-3-one. Levels of 712 -dihydroxy-4-cholesten-3-one, were elevated by 3800-fold in CTX. The measurement of these bile acid precursors represents a much better test for identifying CTX patients than more conventional cholestanol measurements. Dr DeBarber was able to detect elevated levels of 712dihydroxy-4-cholesten-3-one in dried bloodspots collected from newborns with CTX. The method may form the basis for future newborn screening for CTX.

United Leukodystrophy Foundation

Spring 2013

Research/Conference

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Dr. Ingemar Björkhem (Karolinska University, Huddinge, Sweden) discussed the pathogenesis of brain xanthomas in CTX. He noted that the xanthomas in the brain are composed of both cholestanol and cholesterol. Neither of these sterols can pass through the blood-brain barrier (BBB), so their accumulation in xanthomas reflects local synthesis in brain. Dr Björkhem experiments suggest brain xanthomas can develop in CTX due to increased availability of a cholestanol precursor derived from the peripheral circulation. The bile acid precursor 7-hydroxy-4-cholesten-3-one, which is elevated in CTX, can cross the BBB and may be an important precursor for cholestanol synthesis in brain. Dr Björkhem proposed that treatment with CDCA helps resolve brain xanthomas due to suppression of CYP7A1- initiated bile acid synthesis and results in a consequent decrease in blood 7-hydroxy-4cholesten-3-one. Moreover, cholestanol itself increases the rate of cholesterol synthesis, which may contribute to the cholesterol content of the xanthomas. Thus, brain xanthomas develop in CTX because of the increased availability of a cholestanol biosynthetic precursors derived from the peripheral circulation, together with the cholestanol-induced up regulation of brain cholesterol synthesis. Among all leukodystrophies, the treatment of CTX is remarkable for its simplicity and effectiveness. Dr. Gerald Salen (New Jersey Medical School, Newark, NJ) discussed his findings that the metabolic defect in hepatic sterol 27-hydroxylase preventing synthesis of the bile acid end product, CDCA, could be treated by supplementation with CDCA (brand name Chenodal). CDCA that is normally produced regulates bile acid synthesis by suppressing CYP7A1- initiated bile acid synthesis. Impaired synthesis of CDCA in CTX leads to unrestrained CYP7A1-initiated bile acid synthesis and high levels of bile acid precursors like 7hydroxy-4-cholesten-3-one and cholestanol accumulation. Oral administration of CDCA replaces this missing bile acid and down regulates CYP7A1 activity, thereby decreasing bile acid and cholestanol synthesis. Over time, CDCA therapy lowers cholestanol levels in the body, halts the progressive growth of xanthomas and may even improve some symptoms. It is clear, however, that therapy with CDCA is most effective in preventing symptoms rather than reversing them. Dr. James Heubi (University of Cincinnati College of Medicine, Cincinnati, OH) described an alternative therapy for children with CTX using oral cholic acid supplementation, which has no heptotoxicity compared to CDCA and also inhibits production of bile alcohols quite effectively. He has treated two CTX infants with cholic acid and improved their cholestatic liver disease. After 10 years, the patients are doing well on cholic acid alone. For patients with CDCA side effects or in pediatric patients, cholic acid is a potentially useful therapy. Further long-term studies are needed to determine its effectiveness for CTX. ~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~

The Future of Leukodystrophy Therapy is Now! Save the date for the ULF Scientific Meeting & Family Conference. The Scientific Meeting is on Thursday, July 18th and the Family Conference is on Friday, July 19th and Saturday, July 20th. Family registration cost is $200.00 per person for ULF members, children under 18 are free. Annual dues are $35.00 per individual/family and $70.00 for professionals/organizations and are due at the time of registration for the conference, if not yet paid. 2013 annual membership dues notices were recently mailed, your prompt response is greatly appreciated! Confirmed speakers are: Orna Elroy-Stein, Ph.D., Tel Aviv University, Israel, VWM Joseph Hacia, Ph.D., Univ. of California, San Francisco, CA, Zellweger Syndrome Paola Leone, Ph.D., U of Med & Dent of New Jersey, Camden, NJ, Canavans Ann Moser, B.A., Kennedy Krieger Institute, Baltimore, MD, Newborn Screening Aurora Pujol, M.D., Ph.D., Institut d'Investigació Biomedica de Bellvitge, Barcelona, Spain, AMN William Rizzo, M.D., Univ. of Nebraska Medical Center, Omaha, NE, Chair, ULF Medical and Scientific Advisory Board David Rowitch, M.D., Ph.D., Univ. of California, San Francisco, CA, PMD Steve Steinberg, Ph.D., Kennedy Krieger Institute, Baltimore, MD, Peroxisomal Disorders Marjo van der knaap, M.D., Ph.D., VU University medical Center, Amsterdam, The Netherlands, VWM Adeline Vanderver, M.D., Children’s Hospital National Medical Center, Washington, DC, Myelin Disorders Kathleen Zackowski, Ph.D., O.T., Kennedy Krieger Institute, Baltimore, MD, Heterozygotes

Other world renowned leukodystrophy scientists and researchers will also be present. An agenda with the sessions and times will be posted on our website soon. A registration form is included in this newsletter so you can register early and have plenty of time to make travel arrangements to DeKalb, Illinois at reasonable rates.

United Leukodystrophy Foundation

Spring 2013

Family Conference

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The Future of Leukodystrophy Therapy is Now! NO WALK-INS Please Register by Monday July 8, 2013

FAMILY REGISTRATION FORM LEUKODYSTROPHY MEETING July 19th & 20th, 2013 First & Last Name: First & Last Name: Child First & Last Name:

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FAX registration form to: (815) 748-0844 or call (800) 728-5483 - (815) 748-3211 or e-mail: [email protected]

Attendees for the ULF Meeting will pay $200.00 registration fee per person ($100 non-refundable). One day only $100.00 registration fee per person ($50.00 non-refundable). Children no charge. Hotel room costs are to be paid directly to the hotel at $89.99 plus tax per night. 2013 Annual Dues ($35.00) will be added if not yet paid. A registration form must be submitted to the ULF with a check or credit card payable to the ULF. Hotel charges will be separate and paid directly to the hotel. Registration deadline is July 8, 2013. $_______ Registration Fee(s)

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United Leukodystrophy Foundation

Spring 2013

Memorials

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IN MEMORY OF… Ramon Abanilla Ligaya Pollosco Mari Lea Allison Audrey H. Kinney Michael Alm J. Brian & Kathleen Murphy Thomas Andre Gail & David Andre Blake Andrews Mevin & Mamie Andrews Sherri Andrews Frank Beal Rob & Kathy Childers Sabrina Standifer & Duane Hawkes Leroy & Pat Lehman Stephen & Debra Lewis Dustin Looney Brian & Lisa McCoy Sandra Otto Wayne & Carolyn Ray Frances Jo-An Rayburn Melvin Leif Rayburn Gail & Stan Slater Haley Slater Allen & Lenora Wilson Phillip Andrews Melvin & Mamie Andrews William Armstrong Fortuna & Steven Scheige Jacob Townsend Ayers Cicero & Alice Ayers Thomas Baldwin Gerald & Merlee Adkins Richard & Deana Everill Maryellen Garrison Herman & Carol Miller Amity & Paul Noltemeyer Mary Ruter Ellen Borchers Alfred & Mildred Borchers Kim Borchers Alfred & Mildred Borchers Howard Brazeal Patrick & Pam Mumey Paula Brazeal Patrick & Pam Mumey Ron Brazeal Patrick & Pam Mumey Timmy Brazeal Patrick & Pam Mumey Arnold Brown Leslie Sisco David Brown Lee & Martha Hamilton Bob & Anne Siress Linda Brown Lee & Martha Hamilton Bob & Anne Siress George Buckner Sally Buckner Dorothy Busker Butch, Colleen & Matt Johnson

Vinny Capozzi Hugh & Kathryn Aberman Alert Pharmacy Services Mark & Marion Bittner Lisbeth Capozzi Carlisle Carrier Corporation Jeffrey Cramer Drs. Kearns, Ashby, Rajchel & Assoc. Terry Hegglin Marvin & Joan Hoke Mark & Lisa Hummel InsuranceNewsNet.com, Inc. Steve & Sylvia Kinney LeadingAge PA Maxim Healthcare Services Daniel Natirboff Pickering Manor Patricia Putz Sonya Ranker Ronald Reeder Holly Rosini Richard & Julie Ruda Terri Sherman South Middleton Elementary PTO William F. & Roger M. DeCarbo Funeral Home Nora Winkelman Ziegler & Co. Jessica Carter Debby & Ralph Morris Meagan Casey Timothy Casey Homer Coffman, III Jack & Linda Sheetz Thomas Coxworth Andrea Billhardt Michael Day Jim & Heather Day William Dorn Jody & Rick Vogan Katie Dosal Cindy Howard Andrew Fingeroot Bruce & Bea Nahon Donald Fleming Keith & Martha Yale Kenneth Fleming Keith & Martha Yale Maxwell Foutch Alan & Joann Mitchell Jenna Lee Fulmer Dorothy M. Keller, Esq. Alison Marie Gartig Barbara & Bill Gartig Todd William Gillespie Ken & Geraldine Gillespie Lauren Glenn Thomas & Anne Glenn Samuel Goldman Nidec Motor Corporation Michael Grandazza Luella & Peter Grandazza

United Leukodystrophy Foundation

Ken Griffiths William & Bonnie Bailey George Haines Cheryl Haines Russell Allen Hargrove Donna Parkes John Horvat John & Regina Horvat Russell Horvat John & Regina Horvat Michael Jungwirth Joan Jungwirth Clint Karlsrud Chris & Amy Karlsrud Shane Keeler Donnie & Mickey Mitchell Kelli Kovacs Roberta Johanson Joseph Micheal Kulbacki H. Leigh & Sharon Severance Nicholas Lamendola Kimberly Lamendola Douglas Lewis Orville & Stephanie Lewis Richard Louisell Patti & Larry Chapman James Mahan, Jr. James & Mary Mahan Kelsey R. McDonald Alice Miller Natalie Meadows Garry Meadows Dylan Carl Mercer Ligaya Pollosco Ladd Molin Patrice & Alan Solyntjes Ryan Monaghan Michele & Robert Curtis Jaybird Monroe Ed & Maribeth Monroe Jeffery Monroe Ed & Maribeth Monroe Heather Morris Steve Morris Dr. Hugo Moser Ep Haven Alison Rachael Muller Cynthia & Jay Muller Jessica Naugle Bill & Felicia Glass Daniel O’Brien James & Karen Baumhardt Trace & Cindy Blakely Sister Mary Noel Brown Thomas & Bonita Eiring Mary Hays James & Cynthia Koffman John Korb Faith Krueger Robert & Rose Loehr Red Cabin Hospitality, Inc. Timothy & Jelaine Scannell Thomas & Suzanne Wegner

Kirk Padgett Betty Padgett Trey Padgett Betty Padgett Luka Pelka Robert & Virginia Traynor Talamus B. Perry Delonsaletta Perry Eileen Peterson Laura Gershenson Debbie Sukenic Grace Ragon Kelly Biller Nathan & Marla Chandler Mr. & Mrs. Wilfrid Dues Mr. & Mrs. Kendal Peck Margaret Pennucci Michael & Sherri Warren Timothy & Jill Weber Ronald & Wanda Winkle Kim Raleigh Jerry & Connie Altman Mark & Kim Bradford Marc & Linda Ingraham Dr. Charles Peters Peter & Donna Mae Raleigh Phil & Pat Raleigh Thomas Reising Carol & Jim McGinley Elena Routhier Jeanine & Dennis Routhier Jonathan & Lara Routhier Noah Routhier Jeanine & Denis Routhier Dorothy Russell Nichole & Edward Humes Lawrence & Janice Russell Ronald Sato Alice Sato Christopher Stewart Donald & Bonnie Stewart Alyssa Stramel Tom & Stephanie Garrity Matthew Sundstrom John & Margaret Phillips David Riciotti Mitchell Sundstrom John & Margaret Phillips David Riciotti Nathaniel Kent Thomas Kim Thomas Brenton Troup James & Susie Troup Thomas Isaiah M. Wallace Tenny Henderson

Spring 2013

Honors/Sympathies

IN HONOR OF…

Joseph Abell Dennis & Deborah Benson The Backenson Family Joseph & Christina Conroy Christopher Benson Roxana Habibi Asher Fleming Bloxom Keith & Martha Yale Olivia Borodychuk Chad & Lisa Borodychuk Alex Bouley Dr. & Mrs. Bernard Levine Charna Levine Gregory Brown Mary Wheeler Tory Brown Jeff Brown

7 The Capozzi Family Alert Pharmacy Services Aidan Chapleau Marla & Ron Chapleau Harold Childress Tenny Henderson Chase Comer Tom & Allison Comer Kelly Cooper Patricia & Richard Teachout Nicholas Devoss H. Leigh & Sharon Severance Heather DeWitt Marilyn Boersma Skyler Di Palma Linda Savana Zane Dial Carol Smiskol Noam Dichek Dina Dichek

Barbara Easley Tenny Henderson The Gates Family Deborah Goerlich Kaitlyn Fryman Tammy McAllister Sharpsburg Country Homemakers Matt Gagliardi Joseph & Christina Conroy Cody Gates Deborah Goerlich Janet Grady Ron & Jean Kauffman Connor Groel Chip & Linda Ridewood, III Joshua Haydt Harry & Patty Haydt Anne Hinson Debbie Hoffman

Ethan Jeffcoat James Mahern, Sr. Paul Korth Duane & Kimberly Ostdiek Thomas Kulbacki H. Leigh & Sharon Severance Ryan Miller Alice Kloos Devon Neff Marissa Neff Maher Daniel Perrine Mark & Jennifer Sigmund Allison Pizzica Dr. & Mrs. Al Pizzica Adam Smith Patricia De Majistre Tammy Truman Kathie & Donald Truman

Sympathies Blake Andrews: February 7, 2013, loving son of Sherri and Darrell Andrews and loving brother of Elizabeth Andrews Ballinger, Waco, TX. Thomas J. Baldwin: December 16, 2012, beloved husband of Evelyn Hope (nee Hess) and loving father of Maryellen (Lynn) Garrison and Sharon Baldwin Sittner, Fairfield, OH. Caysen Landry Barnett: 5 yrs old, July 16, 2012, loving son of Amanda Barnett Moody and Dusty Moody and loving grandson of Larry and Cathy Ferguson, Amarillo, TX. Ann Jacobs Biraben: 66 yrs old, March 1, 2013, loving partner of Jack Berlien and loving mother of Lilli Joncas, Alexandria, VA. Nathaniel John Butler: 37 yrs old, February 12, 2013, loving son of Robert Leo and Wanda Jane (Dillon) Butler, loving brother of Chris and Angie Butler and loving father of Jacob Butler-Piqua, West Milton, OH. Vincent “Vinny” Louis Capozzi: 8 yrs old, February 23, 2013, loving son of Louis and Shelly Capozzi, Jr., loving brother of Louis J. Capozzi, III, Victor Louis Capozzi and Liza A. Capozzi, Carlisle, PA. Kathryn Claire Hance: February 22, 2012, loving mother of Karen Hance Duffey, Kathryn Lynn Hance, Kimberly Hance Pulliam and Kristine Hance Dosal, Tampa, FL. Ronald J. Klein: January 27, 2013, loving husband of Nina (nee Scalise) Klein and loving father of Ricky, Saint Louis, MO. Daniel Thomas O’Brien: 51 yrs old, January 2, 2013, loving husband of Patty and loving father of Joshua Daniel O’Brien, Eden, WI. Eileen Peterson: January 6, 2013, loving mother of Nancy Maurer, Sandra Talbird and Kristin Davis, Coppell, TX. Kimberly Julia Raleigh: 28 yrs old, January 13, 2013, loving daughter of Philip A. and Patricia J. (Keiser) Raleigh and loving sister of Jason, Battle Creek, MI. Dorothy Russell: 87 yrs old, February 26, 2013, beloved wife of Elmer W. Russell and loving mother of Sandra (Keith) Madden, Sharon Spinneweber and Linda (Scott) Houston, Pleasant Hills, PA. Georgina Wyse: March 27, 2011, loving daughter of Madeline and Ian Wyse, United Kingdom.

United Leukodystrophy Foundation

Spring 2013

You Are Not Alone

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