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	<title>About Sanfilippo syndromeAbout Sanfilippo syndrome</title>
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	<link>http://www.curesf.org</link>
	<description>Work.  Pray.  Love.</description>
	<lastBuildDate>Mon, 20 Feb 2012 17:37:57 +0000</lastBuildDate>
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		<title>Informal update on MLD gene therapy clinical trial</title>
		<link>http://www.curesf.org/2012/02/informal-update-on-mld-gene-therapy-clinical-trial/</link>
		<comments>http://www.curesf.org/2012/02/informal-update-on-mld-gene-therapy-clinical-trial/#comments</comments>
		<pubDate>Sun, 19 Feb 2012 14:17:49 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[current events]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7425</guid>
		<description><![CDATA[The site of the MLD Foundation has recently updated their news with an informal report on the gene therapy clinical trial of MLD patients being conducted by the San Raffaele Telethon Institut for Gene Therapy. The trial is part of a strategic alliance between GlaxoSmithKline PLC (GSK), Fondazione Telethon and Fondazione San Raffaele to research &#8230; <a class="more-link" href="http://www.curesf.org/2012/02/informal-update-on-mld-gene-therapy-clinical-trial/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/informal-update-on-mld-gene-therapy-clinical-trial/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
		</item>
		<item>
		<title>Participating in the U.S. Rare Disease Registry (CoRDS)</title>
		<link>http://www.curesf.org/2012/02/rare-disease-registry-at-sanford-cords/</link>
		<comments>http://www.curesf.org/2012/02/rare-disease-registry-at-sanford-cords/#comments</comments>
		<pubDate>Sun, 19 Feb 2012 12:55:12 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[current events]]></category>
		<category><![CDATA[education]]></category>
		<category><![CDATA[MPS research]]></category>
		<category><![CDATA[rare disease]]></category>
		<category><![CDATA[Rare diseases]]></category>
		<category><![CDATA[registry]]></category>
		<category><![CDATA[Sanford research]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7408</guid>
		<description><![CDATA[The Coordination of Rare Diseases at Sanford (CoRDS) registry is a United States disease registry that includes all rare diseases. The CoRDS registry is headquartered at Sanford Research in Sioux Falls, South Dakota and is supervised by Dr. David Pearce and Dr. Chun-Hung Chan and managed by Liz Donohue and Lauren Beaumont.  If you want to enroll your child in the registry, this post contains the information and links to tell you what you need to know. <a class="more-link" href="http://www.curesf.org/2012/02/rare-disease-registry-at-sanford-cords/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/rare-disease-registry-at-sanford-cords/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
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		<item>
		<title>Genzyme announces 4-year results of oral therapy for Gaucher disease</title>
		<link>http://www.curesf.org/2012/02/genzyme-announces-4-year-results-of-oral-therapy-for-gaucher-disease/</link>
		<comments>http://www.curesf.org/2012/02/genzyme-announces-4-year-results-of-oral-therapy-for-gaucher-disease/#comments</comments>
		<pubDate>Thu, 16 Feb 2012 18:03:38 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[featured content]]></category>
		<category><![CDATA[news wire]]></category>
		<category><![CDATA[treatment news]]></category>
		<category><![CDATA[enzyme replacement therapy]]></category>
		<category><![CDATA[gaucher disease]]></category>
		<category><![CDATA[genzyme]]></category>
		<category><![CDATA[Lysosomal Storage Disease]]></category>
		<category><![CDATA[Sanofi]]></category>
		<category><![CDATA[therapy]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7379</guid>
		<description><![CDATA[CAMBRIDGE, Mass.&#8211;(BUSINESS WIRE)&#8211;Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today four-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate. Sustained or further improvements were observed across all endpoints, including markers of bone disease, at the &#8230; <a class="more-link" href="http://www.curesf.org/2012/02/genzyme-announces-4-year-results-of-oral-therapy-for-gaucher-disease/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/genzyme-announces-4-year-results-of-oral-therapy-for-gaucher-disease/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
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		<item>
		<title>Gene therapy for Lysosomal Storage Diseases</title>
		<link>http://www.curesf.org/2012/02/gene-therapy-for-lysosomal-storage-diseases/</link>
		<comments>http://www.curesf.org/2012/02/gene-therapy-for-lysosomal-storage-diseases/#comments</comments>
		<pubDate>Thu, 16 Feb 2012 13:14:50 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[featured content]]></category>
		<category><![CDATA[MPS research]]></category>
		<category><![CDATA[treatment news]]></category>
		<category><![CDATA[adeno-associated virus]]></category>
		<category><![CDATA[Batten disease]]></category>
		<category><![CDATA[enzyme replacement therapy]]></category>
		<category><![CDATA[fabry disease]]></category>
		<category><![CDATA[gaucher disease]]></category>
		<category><![CDATA[Gene therapy]]></category>
		<category><![CDATA[lysosomal storage disorders]]></category>
		<category><![CDATA[metachromatic leukodystrophy]]></category>
		<category><![CDATA[MPS]]></category>
		<category><![CDATA[pompe disease]]></category>
		<category><![CDATA[sanfilippo syndrome]]></category>
		<category><![CDATA[Sanfilippo Type A]]></category>
		<category><![CDATA[serotype 9]]></category>
		<category><![CDATA[vector]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7365</guid>
		<description><![CDATA[Over the years many cell and gene therapy approaches have been tested in animal models of lysosomal storage diseases, and two main approaches have emerged as the most promising for translation into human clinical trials: In vivo gene transfer by direct infusion of viral vectors (AAV and lentivirus vectors) encoding normal enzymes; or modification of bone marrow stem cells in culture with viral vectors encoding normal enzymes (retrovirus and lentivirus vectors) followed by transplantation (this latter approach is often referred to as ex vivo gene therapy). <a class="more-link" href="http://www.curesf.org/2012/02/gene-therapy-for-lysosomal-storage-diseases/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/gene-therapy-for-lysosomal-storage-diseases/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
		</item>
		<item>
		<title>Drug reverses Huntington&#8217;s motor skills damage in animals</title>
		<link>http://www.curesf.org/2012/02/drug-reverses-huntingtons-motor-skills-damage-in-animals/</link>
		<comments>http://www.curesf.org/2012/02/drug-reverses-huntingtons-motor-skills-damage-in-animals/#comments</comments>
		<pubDate>Tue, 14 Feb 2012 16:48:56 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[treatment news]]></category>
		<category><![CDATA[animal models]]></category>
		<category><![CDATA[genetic disease]]></category>
		<category><![CDATA[hereditary disease]]></category>
		<category><![CDATA[huntington disease]]></category>
		<category><![CDATA[neurological disease]]></category>
		<category><![CDATA[neurological disorders]]></category>
		<category><![CDATA[Parkinsons disease]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7343</guid>
		<description><![CDATA[A drug called GM1, being tested to treat Parkinson&#8217;s disease and related neurodegenerative maladies, has successfully restored motor skills in an animal model of Huntington disease during laboratory preclinical testing, at least for a few weeks after the dosing stopped. GM1, a molecule produced in the body, is a type of lipid and is part &#8230; <a class="more-link" href="http://www.curesf.org/2012/02/drug-reverses-huntingtons-motor-skills-damage-in-animals/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/drug-reverses-huntingtons-motor-skills-damage-in-animals/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
		</item>
		<item>
		<title>Intrathecal enzyme replacement to treat MPS VI</title>
		<link>http://www.curesf.org/2012/02/7319/</link>
		<comments>http://www.curesf.org/2012/02/7319/#comments</comments>
		<pubDate>Mon, 13 Feb 2012 20:56:48 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[MPS research]]></category>
		<category><![CDATA[treatment news]]></category>
		<category><![CDATA[Biomarin]]></category>
		<category><![CDATA[enzyme replacement t]]></category>
		<category><![CDATA[galsulfase]]></category>
		<category><![CDATA[Maroteaux-Lamy syndrome]]></category>
		<category><![CDATA[MPS disease]]></category>
		<category><![CDATA[MPS VI]]></category>
		<category><![CDATA[Rare diseases]]></category>
		<category><![CDATA[rhASB]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7319</guid>
		<description><![CDATA[What is MPS VI? MPS VI (mucopolysaccharidosis VI), also known as Maroteaux-Lamy Syndrome, is an inherited lysosomal storage disorder caused by the deficiency of arylsulfatase B, an enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). If the enzyme is not present in large enough amounts, the normal breakdown of &#8230; <a class="more-link" href="http://www.curesf.org/2012/02/7319/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/7319/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
		</item>
		<item>
		<title>Team Jazzy pedals across the UK</title>
		<link>http://www.curesf.org/2012/02/team-jazzy-pedals-across-the-uk/</link>
		<comments>http://www.curesf.org/2012/02/team-jazzy-pedals-across-the-uk/#comments</comments>
		<pubDate>Mon, 13 Feb 2012 20:15:19 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[current events]]></category>
		<category><![CDATA[featured content]]></category>
		<category><![CDATA[online resources]]></category>
		<category><![CDATA[upcoming events]]></category>
		<category><![CDATA[England]]></category>
		<category><![CDATA[Gloucester]]></category>
		<category><![CDATA[jasmin heap]]></category>
		<category><![CDATA[justgiving]]></category>
		<category><![CDATA[team jazzy]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7357</guid>
		<description><![CDATA[What Dave Heap, Tom Appleby &#038; Richard Bartley are childhood friends &#8211; they are getting together on 4th May 2012 to cycle from Lands End to John O&#8217;Groats – a whooping 980 miles, in order to raise much needed funds for Acorns Childrens Hospice and Alderhey Childrens Hospital, Liverpool. They will be pedalling with Jasmin &#8230; <a class="more-link" href="http://www.curesf.org/2012/02/team-jazzy-pedals-across-the-uk/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/team-jazzy-pedals-across-the-uk/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
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		<item>
		<title>Inclusion criteria available for MPS 3B Natural History Study</title>
		<link>http://www.curesf.org/2012/02/inclusion-criteria-available-for-mps-3b-natural-history-study/</link>
		<comments>http://www.curesf.org/2012/02/inclusion-criteria-available-for-mps-3b-natural-history-study/#comments</comments>
		<pubDate>Sun, 12 Feb 2012 02:35:32 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[disease news]]></category>
		<category><![CDATA[featured content]]></category>
		<category><![CDATA[MPS research]]></category>
		<category><![CDATA[clinical trials]]></category>
		<category><![CDATA[enzyme replacement]]></category>
		<category><![CDATA[enzyme replacement therapy]]></category>
		<category><![CDATA[MPSIIIb]]></category>
		<category><![CDATA[natural history study]]></category>
		<category><![CDATA[Shire]]></category>
		<category><![CDATA[type b]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7303</guid>
		<description><![CDATA[The website clinicaltrials.gov has the complete description of the Natural History Study for MPS 3B. Study Dates The study start date is scheduled to be February, 2012. Study Completion for data collection will be August, 2014, with final completion in September of 2014. Study Description As nearly everyone knows, the study involves no treatment. The &#8230; <a class="more-link" href="http://www.curesf.org/2012/02/inclusion-criteria-available-for-mps-3b-natural-history-study/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/inclusion-criteria-available-for-mps-3b-natural-history-study/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
		</item>
		<item>
		<title>Purple Stands for Courage.  The Purple Lemonade Crusade</title>
		<link>http://www.curesf.org/2012/02/the-purple-lemonade-stand-supporting-blair-and-her-friends/</link>
		<comments>http://www.curesf.org/2012/02/the-purple-lemonade-stand-supporting-blair-and-her-friends/#comments</comments>
		<pubDate>Wed, 08 Feb 2012 14:03:48 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[featured content]]></category>
		<category><![CDATA[online resources]]></category>
		<category><![CDATA[stories]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7264</guid>
		<description><![CDATA[Purple Stands for Courage:  Caroline in Fort Lauderdale is hosting a Purple Lemonade Stand for their friend Blair (who has Sanfilippo syndrome), during the Rio Vista Golf Cart Brigade this Saturday, February 11 from 3-6pm at the Rio Vista Park.  They will be selling purple lemonade and baked goods all in a purple theme.
 <a class="more-link" href="http://www.curesf.org/2012/02/the-purple-lemonade-stand-supporting-blair-and-her-friends/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/the-purple-lemonade-stand-supporting-blair-and-her-friends/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
		</item>
		<item>
		<title>U.S. MPS Society issues call for MPS III research idea letters</title>
		<link>http://www.curesf.org/2012/02/u-s-mps-society-issues-call-for-mps-iii-research-idea-letters/</link>
		<comments>http://www.curesf.org/2012/02/u-s-mps-society-issues-call-for-mps-iii-research-idea-letters/#comments</comments>
		<pubDate>Tue, 07 Feb 2012 19:44:14 +0000</pubDate>
		<dc:creator>Elaine</dc:creator>
				<category><![CDATA[current events]]></category>

		<guid isPermaLink="false">http://www.curesf.org/?p=7251</guid>
		<description><![CDATA[MPS Society issues request for Letter of Intent for MPS III Grand Challenge Grant The U.S. MPS Society has issued an RFI in the form of Letters of Intent for MPS III research. The following information is taken from the MPS society website. Deadline for submissions is March 15. Grants are also available for MPS &#8230; <a class="more-link" href="http://www.curesf.org/2012/02/u-s-mps-society-issues-call-for-mps-iii-research-idea-letters/">Continue reading <span class="meta-nav">&#8594;</span></a>]]></description>
		<wfw:commentRss>http://www.curesf.org/2012/02/u-s-mps-society-issues-call-for-mps-iii-research-idea-letters/feed/</wfw:commentRss>
		<slash:comments>0</slash:comments>
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