21. ERT Update mps iii TKT, as part of their research to evaluate new approaches to the problem of treatment of the CNS, is hoping to move their mps iiiA programme forward http://www.mpssociety.co.uk/ertupdate.htm

Society for Mucopolysaccharide Diseases 46 Woodside Road, Amersham, Buckinghamshire, HP6 6AJ, UK Tel: 0845 389 9901 Fax: 0845 389 9902 National Registered Charity No. 287034 Home Diseases Advocacy Support Information Resource ... Terms and Conditions Enzyme Replacement Therapy Update MPS I Aldurazyme is administered once-weekly and has been approved in the European Union for long term enzyme replacement therapy in patients with a confirmed diagnosis of MPS I, to treat the non-neurological manifestations of the disease. As the first orphan drug approved for MPS I in the European Union, Aldurazyme has been granted ten years of market exclusivity. In 2004 some MPS I patients in the United States of America started to receive their once-weekly infusion at home. This has initiated considerable interest by UK MPS I ERT patients having to travel long distances to hospital and spending many hours in a hospital environment. The difficulty is that Aldurazyme was licensed in the European Union for hospital use only. The MPS Society is in dialogue with Genzyme Corporation to see if it is time now for the license to be expanded to include home treatment. MPS II Transkaryotic Therapies Inc (TKT) released research findings on 28 October 2004 at the American Society of Human Genetics 54th Annual Meeting held in Toronto, Canada, evaluating intrathecal delivery of iduronate-2-sulfatase (12S) in an animal model. The results, presented by Dr Justin Lamsa of TKT showed that repeated injections of 12S were well-tolerated and resulted in the accumulation of enzyme in various cells of the central nervous system (CNS).

22. MPS III - Sanfilippo - Healthfinder® A consumer health information minifact sheet about this birth defect discusses causes, types and diagnosis. Sanfilippo is one of the mucopolysaccharide http://mentalhealth.about.com/library/h/docs/bld02351.htm

zJs=10 zJs=11 zJs=12 zJs=13 zc(5,'jsc',zJs,9999999,'') zfs=0;zCMt='a14' About Mental Health Resources Mental Health Essentials ... Help zau(256,140,140,'el','http://z.about.com/0/ip/417/C.htm','');w(xb+xb+' ');zau(256,140,140,'von','http://z.about.com/0/ip/496/6.htm','');w(xb+xb); FREE Newsletter Sign Up Now for the Mental Health Resources newsletter! See Online Courses Search Mental Health Resources MPS III - Sanfilippo internet URL http://www.mpssociety.org/mps3.htm sponsoring agency National MPS Society, Inc. description A consumer health information mini-fact sheet about this birth defect discusses causes, types and diagnosis. Sanfilippo is one of the mucopolysaccharide disorders. Keywords: Birth Defect Consumer Resources From Leonard Holmes, Ph.D. Your Guide to Mental Health Resources FREE Newsletter. Sign Up Now! zau(256,420,100,'ri','http://z.about.com/5/o/c.htm?gs='+gs,'') Most Popular Video Bathroom Decoration Ideas Tag Sale Tips Creating a Craft Room Making a Personal Space ... See other videos at About.com Advertisement Most Popular MAO Inhibitors Cymbalta gets Mixed Reviews Video Game Violence Psychiatric Medications - Introduction, Antipsychotics

23. Entrez PubMed Clinical heterogeneity for Sanfilippo B syndrome (mps iii B) in the same family has never been repor http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=1

24. MPS III - Computing Reference - ELook.org Solving matrices and producing reports. mps iii DATAFORM User Manual , Management Science Sy. Previous Terms, Terms Containing mps iii, Next Terms http://www.elook.org/computing/mps-iii.htm

eLook Home eLook Computing Reference Search Computing Reference More Resources Information US City Data Worldwide Weather Entertainment Video Game Cheats Food Nutrition Recipes Literature Literature Reference Computing Dictionary Internet Encyclopedia Programming Reference ... Thesaurus By Letter: Non-alphabet A B C ... Z MPS III Solving matrices and producing reports. "MPS III DATAFORM User Manual", Management Science Sy Terms Containing MPS III MPP MPPE MPPL MPR II ... Contact

25. Mucopolysaccharidoses mps iii (Sanfilippo syndrome). mps iii, like the other MPS conditions, was initially Many children with mps iii will develop seizures, sleeplessness, http://www.healthatoz.com/healthatoz/Atoz/ency/mucopolysaccharidoses.jsp

26. Ben's Dream - UNC Grant Summary Gene therapy research on Sanfilippo B Syndrome (mps iii B) directed by Dr. This mps iii B gene therapy research at UNC has been possible because of a http://www.bensdream.org/uncgrant.html

Research Home University of North Carolina - Chapel Hill, Dr. Joseph Muenzer Although, we have not succeeded in proving that the enzyme produced after the injection of AAV vectors into the brain of animals corrects the GAG storage, studies with primary mouse brain cell cultures are very encouraging. We have been able to establish primary cultures of MPS III B mouse brain, kidney, liver and skin fibroblast cells. The AAV gene therapy vectors, when added to the cell cultures, are capable of producing the NaGlu enzyme in these MPS III B primary cell cultures, including brain cell, and the storage of GAG is corrected. In addition, AAV expressed enzyme is secreted by the cultured brain cells into the culture media. This secreted enzyme is also able to correct the storage of GAG when added to MPS III B mouse brain cells in culture. These experiments support the concept that AAV vectors can deliver the MPS III B human gene to brain cells, and the enzyme produced will correct the storage of GAG in the central nervous system.

27. HONselect - Mucopolysaccharidosis III mps iii A mps iii B - mps iii C - mps iii D. Français, Mucopolysaccharidose de type III, - Maladie de Sanfilippo - Mucopolysaccharidose de San Filipo http://www.hon.ch/HONselect/RareDiseases/C17.300.550.575.650.html

List of rare diseases: English Deutsch Language: MeSH term: Accepted terms: English: Mucopolysaccharidosis III - Polydystrophic Oligophrenia - Sanfilippo's Syndrome - MPS III A - MPS III B - MPS III C - MPS III D Français: Mucopolysaccharidose de type III - Maladie de Sanfilippo - Mucopolysaccharidose de San Filipo - Mucopolysaccharidose de Sanfilippo - Mucopolysaccharidose de type 3 - Oligophrénie polydystrophique de Lamy-Maroteaux Deutsch: Mukopolysaccharidose Typ III - Polydystrophische Oligophrenie - Sanfilippo-Krankheit Español: Mucopolisacáridosis III - Oligofrenia Polidistrófica - Síndrome de Sanfilippo Português: Mucopolissacaridose III - Oligofrenia Polidistrófica - Síndrome de Sanfilippo HONselect ressources Definition: Yes Articles: Yes Images: No News: No Conferences: No Clinical trials: No Web sites: English Yes Français Yes Deutsch No Español No Português No Home About us Site map Search ... Contact http://www.hon.ch/HONselect/RareDiseases/C17.300.550.575.650.html Last modified: Wed May 18 2005

28. Mucopolysaccharidosis III - Polydystrophic Oligophrenia - Information Page With Synonym(s) Polydystrophic Oligophrenia / Sanfilippo s Syndrome / mps iii A / mps iii B / mps iii C / mps iii D / Mucopolysaccharidosis 3 / San Filippo s http://www.hon.ch/HONselect/RareDiseases/EN/C17.300.550.575.650.html

InitBulle("navy","#F8F8F8","#000066",1); HONcode sites All Web sites HONselect News ... Images HONselect Search English French German Spanish Portuguese the word the part of word in MeSH term in MeSH term and description Information on "Mucopolysaccharidosis III": Medical hierarchy and definition Research Articles Web resources Medical Images Medical News Medical Conferences Clinical Trials Hierarchy English French German Spanish Portuguese Mucopolysaccharidosis III Definition: Mucopolysaccharidosis characterized by heparitin sulfate in the urine, progressive mental retardation, mild dwarfism, and other skeletal disorders. There are four clinically indistinguishable but biochemically distinct forms, each due to a deficiency of a different enzyme. Synonym(s): Polydystrophic Oligophrenia / Sanfilippo's Syndrome / MPS III A / MPS III B / Browse New search Web resources for "Mucopolysaccharidosis III" English = Site with HON description - = Site with a robot description info: enter the site: (click below) domain of the site: Introduction: Mucopolysaccharidosis III - WrongDiagnosis.com www.wrongdiagnosis.com HONcode - www.emedicine.com

29. Department Of Human Genetics, School Of Medicine, Emory University : Ask The Gen In the disease mps iii Sanfilippo Syndrome, the enzyme missing that causes the Mucopolysaccharidosis type III (mps iii) or Sanfilippo syndrome is a http://server2k.genetics.emory.edu/ask/user/view_response.pl?resp=156&temp=3

30. Glycosaminoglycans And Proteoglycans mps iii A Sanfilippo A, Heparan Nsulfatase, heparan sulfate mps iii C Sanfilippo C, AcetylCoA a-glucosaminide-acetyltransferase, heparan sulfate http://web.indstate.edu/thcme/mwking/glycans.html

Glycosaminoglycans Characteristics of GAGs Proteoglycans Clinical Significances ... Return to Medical Biochemistry Page Glycosaminoglycans The most abundant heteropolysaccharides in the body are the glycosaminoglycans (GAGs) . These molecules are long unbranched polysaccharides containing a repeating disaccharide unit. The disaccharide units contain either of two modified sugars- N-acetylgalactosamine (GalNAc) or N-acetylglucosamine (GlcNAc) and a uronic acid such as glucuronate or iduronate . GAGs are highly negatively charged molecules, with extended conformation that imparts high viscosity to the solution. GAGs are located primarily on the surface of cells or in the extracellular matrix (ECM). Along with the high viscosity of GAGs comes low compressibility, which makes these molecules ideal for a lubricating fluid in the joints. At the same time, their rigidity provides structural integrity to cells and provides passageways between cells, allowing for cell migration. The specific GAGs of physiological significance are hyaluronic acid dermatan sulfate chondroitin sulfate heparin heparan sulfate , and keratan sulfate . Although each of these GAGs has a predominant disaccharide component (see Table below), heterogeneity does exist in the sugars present in the make-up of any given class of GAG.

31. Society MPS How common is mps iii? How are the disorders inherited? Prenatal diagnosis Clinical problems in mps iii Physical Appearance Runny nose http://www.mps.sart.pl/htm/en_text/index.php?dzial=mps_3_a

32. Society MPS mps iii is characterized by developmental delay, with usually mild physical problems. Scientists who study mps iii continue to look for better and more http://www.mps.sart.pl/htm/en_text/index.php?dzial=mps_3_b

33. Frankiepants.com On March 17, 2005, our son, Frankie was diagnosed with mps iii Type A. mps iii Type A, From the MPS Society web site, mps iii is described as follows http://www.frankiepants.com/

34. Journal Of Computer Assisted Tomography - UserLogin We describe the cranial findings in two patients with mps iii (Sanfilippo Serum analysis determined a diagnosis of mps iii type B. This patient did not http://www.jcat.org/pt/re/jcat/fulltext.00004728-199711000-00009.htm

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35. Sanfilippos Sjukdom - Små Och Mindre Kända Handikappgrupper mps iii. Innehåll. Sjukdom/skada/diagnos Förekomst Orsak till sjukdomen/skadan Ärftlighet Symtom Diagnostik Behandling/åtgärder Praktiska tips http://www.sos.se/smkh/1997-29-048/1997-29-048.htm

Socialstyrelsen 106 30 Stockholm Socialstyrelsen klassificerar sin utgivning i olika dokumenttyper Sanfilippos sjukdom MPS III Sjukdom/skada/diagnos Orsak till sjukdomen/skadan Symtom Diagnostik ... Dokumentinformation Dokumentdatum: 2004-01-12 HTML-version: 3.0 Socialstyrelsen Detta är ett utdrag ur Socialstyrelsens kunskapsdatabas om små och mindre kända handikappgrupper. Med små och mindre kända handikappgrupper avses ovanliga sjukdomar/skador som leder till omfattande funktionshinder och som finns hos högst 100 personer per miljon invånare. Syftet med databasen är att ge aktuell information om små och mindre kända handikappgrupper och om det stöd och den service som dessa grupper behöver. För ytterligare information om aktuell diagnos hänvisas till informationsmaterial, litteratur och databaser som anges under resp diagnos. Sjukdom/skada/diagnos Orsak till sjukdomen/skadan Symtom Diagnostik Praktiska tips Resurspersoner Huddinge Universitetssjukhus, 141 86 Stockholm Universitetssjukhuset i Lund, 221 85 Lund : Professor Paul Uvebrant.

36. Mucopolysaccharidosis HurlerScheie MPS II - Hunter mps iii - Sanfilippo MPS IV - Morquio MPS VI - Maroteaux - Lamy ML-II and ML III The History of the MPS Society E-mail http://www.health-nexus.com/mucopolysaccharidosis.htm

The #1 Health information site Health-Nexus.Net Health-Nexus.Org Home ... Up Search Health-Nexus for: Match ALL words Match ANY word Email this page to a friend ! Post a question or comment on our Message Board Home Page Health Specialties Health News ... Alternative Health Options Substance Abuse Animal Health Search: Books Magazines Video Keywords: Find it Here Mucopolysaccharidosis Mucopolysaccharidosis Mucopolysaccharidosis conditons international support groups, clinics with genetic counselors and geneticists. ... Mucopolysaccharidosis Syndromes. ... National MPS Society The National MPS Society is a non-profit organanization advocating for families affected by the Mucopolysaccharide diseases. ... NLM - Mucopolysaccharidosis (MPS) I Uncover a comprehensive list of the various synonyms of the rare disease, read a short summary, and learn about its major clinical features. eMedicine - Mucopolysaccharidosis Type IH : Article by Donald ... Mucopolysaccharidosis Type IH - Mucopolysaccharidoses (MPSs) are a group of 7 inherited lysosomal storage diseases caused by a deficiency in the lysosomal ... eMedicine - Mucopolysaccharidosis Type III : Article Excerpt by: Donald Nash, PhD

37. Phorum - OLD Canadian MPS Forum - MPS AND AUTISM / AUTISTIC SYMPTOMS How does one confirm the mps iii diagnosis, and what are the tests which can confirm that the patient has MPS, and more specifically mps iii? http://www.mpssociety.ca/phorum/read.php?f=1&i=39&t=39

38. MPS III More information on mps iii is available from our National Office, including an mps iii syndrome booklet which can be purchased with the attached order form http://www.mpssociety.org.au/MPS Diseases/mps_iii.htm

Home MPS Society MPS Diseases Contact Us ... Site Map MPS III - Sanfilippo Characteristics of MPS III Medical Problems of those affected by MPS III Current Research into MPS III Sanfilippo Syndrome is also known as MPS III. It takes its name from Dr. Sylvester Sanfilippo who was one of the doctors in the United States who described the condition in 1963. To date four different enzyme deficiencies have been found to cause Sanfilippo Syndrome and so the condition is described as type A, B, C, or D. These enzymes are: Sanfilippo A Heparan-N-sulphatase Sanfilippo B a -N-Acetylgucosaminidase Sanfilippo C AcetylCoA:N-acetyltransferase Sanfilippo D N-Acetylglucosamine 6-sulphatase There is usually very little difference between the four types of the disorder but there have been some very mild cases of the B form where the affected individuals have remained relatively healthy into adult life. The latest understanding is that some people seem to produce some enzyme activity which helps to slow down the progression of the disorder whilst those with more severe symptoms appear to have no enzyme activity (or function) at all. It is important to understand that all those with Sanfilippo syndrome have the same condition, even if some have a milder form. The combined incidence of Sanfilippo syndrome is 1 in 66,000, however each of the particular types is rarer again. The Australian prevalence of MPS IIIA is 1 in 114,000, MPS IIIB is 1 in 211,000, MPS IIIC is 1 in 1,407,000 and MPS IIID is 1 in 1,056,000.

39. CharaterIII mps iii is a disorder largely characterised by neurological deterioration. Although babies with mps iii usually appear normal at birth, http://www.mpssociety.org.au/MPS Diseases/charateriii.htm

Home MPS Society MPS Diseases Contact Us ... Site Map Characteristics of MPS III MPS III is a disorder largely characterised by neurological deterioration. Although babies with MPS III usually appear normal at birth, over time characteristic symptoms begin to appear such as: Behavioural problems Sleep disturbance Loss of skills such as speech and learning Frequent respiratory infections Mental retardation in the severe forms Bone deformities and joint stiffness Epilepsy Sanfilippo children grow to a fairly normal height and changes in appearance may be less than in other MPS diseases. The hair is thick and coarser than usual and their bodies may be hairier than normal. The eyebrows are often dark and bushy and may meet in the middle. Noses tend to be upturned and flat on the bridge. The disorder will affect children differently and its progress will be much faster in some cases than in others. Change will usually be very gradual and therefore easier to adjust to. The disorder tends to have three main stages. The first during the child's pre-school years may be a very frustrating one for the parents. They begin to worry as their child starts to lag behind their friend's children in development and they may feel they are being blamed for the child's overactive and difficult behaviour.

40. Complementary And Alternative Therapies For MPS III (Sanfilippo Syndrome) Complementary and Alternative Therapies for mps iii (Sanfilippo Syndrome) http://www.overcome-diabetes.com/th-diabetic/mps3/mps3-index.htm

Your browser does not support inline frames or is currently configured not to display inline frames. Complementary and Alternative Therapies for MPS III (Sanfilippo Syndrome) The Best Complementary and Alternative Therapies for MPS III (Sanfilippo Syndrome) Dietary changes that may be helpful for MPS III ( sanfilippo syndrome ) Chewing As they become more out of touch with their environment, many children with Sanfilippo syndrome will entertain themselves by rocking or by chewing on their fingers, clothes, or whatever they can lay their hands on. Because there is little one can do to stop this behavior, it is best to provide the individual with a wide range of things on which to chew ¨C rubber toys, teething rings, and large Lego blocks are examples. If the problem is severe and the child starts to injure his or her fingers, it is possible to splint the elbows for periods of the day so that the hands cannot reach the mouth. Physical therapy When the child is young and mobile, physical therapy may not be needed. Chest physiotherapy may be needed later to help clear an infection. As the child gets older, the joints of the feet and ankles may become tight and spastic. Hydrotherapy may be a great help in keeping the joints mobile. Some range-of-motion physical therapy may be useful but need not be intensive. Exercises that cause pain should be avoided. When the child is immobile, it is important to ensure that he or she is sitting with proper support to avoid uneven pressure on particular joints. If a deformity at the ankle joint develops, making walking difficult, special braces may help.

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