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Korean Pharma Compan…
Korean Pharma Company gets approval for Hunter syndrome drug
Posted 8 hours ago

The Korean pharmaceutical company Green Cross has developed and received approval from the Korean FDA for an alternative, licensed therapeutic enzyme to treat Hunter Syndrome, a rare disease that until…

Read More
Genistein in Sanfili…
Genistein in Sanfilippo disease (Abstract, Ann Neurol)
Posted 22 hours ago

Genistein in Sanfilippo disease: A randomized controlled crossover trial.
Authorsde Ruijter J, Valstar MJ, Narajczyk M, Wegrzyn G, Kulik W, Ijlst L, Wagemans T, van der Wal WM, Wijburg FA.
Journal
Ann Neurol.…

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Making a weighted bl…
Is a weighted blanket right for your child?
Posted 4 days ago
Is a weighted blanket right for your child?

Weighted blankets and vests are in widespread use as therapeutic devices for children and adults with sensory conditions, most commonly ADD and autism. Several parents of children with MPS disorders…

Read More
Uncle climbs Mount K…
Uncle climbs Mount Kiliminjaro to honor Sanfi…
Posted 7 days ago
Uncle climbs Mount Kiliminjaro to honor Sanfi…

Patrick Chapin, 44, President of the Winter Park, Florida, Chamber of Commerce, has just completed the adventure climb of a lifetime to honor his niece, Blair, and her compatriots with…

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Apply for MPS Societ…
Apply for MPS Society Continuing Education Sc…
Posted 8 days ago
Apply for MPS Society Continuing Education Sc…

MPS Society Continuing Education Scholarship Program
If you or an immediate (United States) family member have an MPS disorder, you are eligible for an MPS Society scholarship. Here's the info from…

Read More
Fish Oil may help re…
Fish Oil may help regenerate damaged nerves (J of Neuroscien…
Posted 10 days ago

Here's the latest: Researchers at Queen Mary, University of London believe that omega-3 fatty acids can protect nerves from being injured and help regenerate the ones that are harmed, based…

Read More
Shire's MPS 3B Natur…
Shire proposes Natural History study of MPS 3…
Posted 12 days ago
Shire proposes Natural History study of MPS 3…

The clinical trial plan for the U.S. natural history study for Sanfilippo Syndrome Type B has just been published in clinicaltrials.gov. It is essentially the same plan as the current…

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Join Global Genes Pr…
Join Global Genes Project in the Rare Disease…
Posted 18 days ago
Join Global Genes Project in the Rare Disease…

Care and share this story! Help your Facebook friends learn more about Sanfilippo syndrome.
A message from the Global Genes Project
We are calling all bloggers to join us in a…

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Slide 1

chloe key, mps 3a

Slide 2

daniel lopez,5,MPS 3B, madrid,spain

Slide 3

Lucas Hembree, stylin and profilin. Lucas has Sanfilippo syndrome Type A

Slide 4

Lucas Montgomery, Cedar Rapids, Iowa, MPS 3B

Slide 5

Jarod and Caleb mulder, MPS 3A

Slide 6

Waverly McNeil, MPS 3A

Slide 7

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Korean Pharma Company gets approval for Hunter syndrome drug

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The Korean pharmaceutical company Green Cross has developed and received approval from the Korean FDA for an alternative, licensed therapeutic enzyme to treat Hunter Syndrome, a rare disease that until now had only one treatment option available.
Continue reading →

Gallery

Genistein in Sanfilippo disease (Abstract, Ann Neurol)

Posted on by

Genistein in Sanfilippo disease: A randomized controlled crossover trial. Authors de Ruijter J, Valstar MJ, Narajczyk M, Wegrzyn G, Kulik W, Ijlst L, Wagemans T, van der Wal WM, Wijburg FA. Journal Ann Neurol. 2012 Jan;71(1):110-20. doi: 10.1002/ana.22643. Affiliation Department of Pediatrics and Amsterdam Lysosome Centre “Sphinx”, University of Amsterdam, Amsterdam, The Netherlands. Abstract OBJECTIVE … Continue reading →

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Is a weighted blanket right for your child?

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Weighted blankets and vests are in widespread use as therapeutic devices for children and adults with sensory conditions, most commonly ADD and autism. Several parents of children with MPS disorders report that their children benefit from the use of a weighted blanket. Weighted blankets are often used for children with sensory processing disorders, which is … Continue reading →

Gallery

Uncle climbs Mount Kiliminjaro to honor Sanfilippo children

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Patrick Chapin, 44, President of the Winter Park, Florida, Chamber of Commerce, has just completed the adventure climb of a lifetime to honor his niece, Blair, and her compatriots with Sanfilippo syndrome. Patrick wants to help build awareness of the disease, and to encourage efforts to find answers for the children who suffer from it. Patrick took along two special items on his journey: A Courage flag with Blair’s name along with the names of some of her friends who also have Sanfilippo syndrome — and Blair’s favorite character-Barney. Continue reading →

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Apply for MPS Society Continuing Education Scholarships

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MPS Society Continuing Education Scholarship Program If you or an immediate (United States) family member have an MPS disorder, you are eligible for an MPS Society scholarship. Here’s the info from the U.S. MPS Society. 2012 Scholarship Applications are now available! Applications must be received by March 15, 2012. Applicants will be notified of the … Continue reading →

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Fish Oil may help regenerate damaged nerves (J of Neuroscience)

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Here’s the latest: Researchers at Queen Mary, University of London believe that omega-3 fatty acids can protect nerves from being injured and help regenerate the ones that are harmed, based on preclinical research with mice. Omega-3s, of course, are found in fish oil. And beyond the latest finding, scientists are discovering that fish oil has the potential to solve plenty of health problems. Continue reading →

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Shire proposes Natural History study of MPS 3B

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The clinical trial plan for the U.S. natural history study for Sanfilippo Syndrome Type B has just been published in clinicaltrials.gov. It is essentially the same plan as the current Natural History study for Type A. We have reproduced it here and linked to the official clinical trials record. This is further evidence that Shire is proceeding rapidly with a plan to carry out clinical trials of enzyme replacement for MPS 3B.
Continue reading →

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Pol Merchan Zafra, Terrassa, Spain

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Care and share this story! Help your Facebook friends learn more about Sanfilippo syndrome. [fbshare] Handsome Pol lives in Terrassa, Spain, with his family. Pol has Sanfilippo Type C. His mother, Belen, is a founder of Sanfilippo Barcelona. That organization is very active in encouraging and supporting research and awareness of Sanfilippo syndrome. Since Pol … Continue reading →

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Build with the Boyces!

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Brooklyn, 3, and Jayden Boyce, 5, are the children of Stefanie and Justin Boyce. Both were diagnosed with Sanfilippo Syndrome in October, 2009. Stefanie and Justin have dedicated themselves to not only giving their own children the best life possible, but to telling their story and raising funds to build a completely handicap accessible home. … Continue reading →

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Join Global Genes Project in the Rare Disease Blog Hop!!

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Care and share this story! Help your Facebook friends learn more about Sanfilippo syndrome. A message from the Global Genes Project We are calling all bloggers to join us in a RARE disease focused blog hop on Monday, January 30th which will be 30 days before World RARE Disease Day. If you have an MPS-centered … Continue reading →

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Ultragenyx In-Licenses Enzyme Replacement Therapy for MPS 7 from St. Louis Univ

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Press release from Marketwatch, January 5, 2012. MPS 7 Patients May at Last have an Opportunity for Enzyme Replacement Therapy NOVATO, Calif., Jan. 5, 2012 /PRNewswire via COMTEX/ — Ultragenyx Pharmaceutical, Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced it has in-licensed an enzyme replacement therapy program … Continue reading →