Gaucher's Disease
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Gaucher's disease is a rare, inherited disorder that causes too much of a substance called glucocerebroside to build up in your spleen, liver, lungs, bones and sometimes in your brain. The buildup prevents these organs from working properly.
There are three types:
- Type 1, the most common form, causes liver and spleen enlargement, bone pain and broken bones, and, sometimes, lung and kidney problems. It does not involve the brain. It can occur at any age.
- Type 2, which causes severe brain damage, appears in infants. Most children who have it die by age 2.
- In type 3, there may be liver and spleen enlargement, and signs of brain involvement appear gradually.
Gaucher's disease has no cure. Treatment options for types 1 and 3 include medicine and enzyme replacement therapy, which is usually very effective. There is no good treatment for the brain damage of type 2.
NIH: National Institute of Neurological Disorders and Stroke
Provided by: NIH - National Institute of Health
Links shared publicly online about this topic
NGF - Gaucher Disease Awareness Month
gaucherdisease.org — “My name is Karina V?squez Fuentes. I am 29 and I suffer from Gaucher Disease” View full resource at gaucherdisease.org
Most Recently Shared on October 28, 2011 at 2:30 pm By:
October is Gaucher Awareness Month. To better understand this rare disease, read Karina Vásquez Fuentes’s story: http://t.co/Gq5WZBnJ
Gaucher disease: FDA approves VPRIV
news-medical.net — “The U.S. Food and Drug Administration has approved velaglucerase alfa for injection (VPRIV) to treat children and adults with a form of the rare genetic disorder Gaucher disease.” View full resource at news-medical.net
Most Recently Shared on February 27, 2010 at 4:57 am By:
Stroke News: Gaucher disease: FDA approves VPRIV: The U.S. Food and Drug Administration has approved velaglucera... http://twurl.nl/zaidct
Elelyso Approved for Gaucher Disease --Doctors Lounge
doctorslounge.com — “Elelyso Approved for Gaucher Disease --Doctors Lounge” View full resource at doctorslounge.com
Most Recently Shared on May 2, 2012 at 4:14 am By:
Elelyso Approved for Gaucher Disease http://t.co/Xbc54SZp #health #medicine
Vpriv available for Type 1 Gaucher disease - ONA
oncologynurseadvisor.com — “Shire announced that Vpriv (velaglucerase alfa for injection) has been FDA approved and made available for the treatment of Type 1 Gaucher disease.” View full resource at oncologynurseadvisor.com
Most Recently Shared on March 1, 2010 at 4:13 pm By:
Vpriv available for Type 1 Gaucher disease http://goo.gl/fb/3kQb
FDA Approves VPRIV to Treat Gaucher Disease
drugs.com — “VPRIV (velaglucerase alfa) is used to treat Gaucher disease. Includes VPRIV side effects, interactions and indications.” View full resource at drugs.com
Most Recently Shared on February 28, 2010 at 5:02 am By:
FDA Approves VPRIV to Treat Gaucher Disease - http://bit.ly/dATgn7
Gastro Medical News about Gaucher Disease
mdlinx.com — “The US Food and Drug Administration Approves a New Gaucher Disease Drug” View full resource at mdlinx.com
Most Recently Shared on June 8, 2010 at 7:54 pm By:
The US Food and Drug Administration Approves a New Gaucher Disease Drug: Gastroenterology http://bit.ly/d1S9dg #GI
FDA Approves Therapy To Treat Gaucher Disease
tweetmeme.com — “The U.S. Food and Drug Administration has approved velaglucerase alfa for injection (VPRIV) to treat children and adults with a form of the rare genetic disorder Gaucher disease. Gaucher disease occurs in people who do not produce enough of an enzy” View full resource at tweetmeme.com
Most Recently Shared on February 27, 2010 at 2:49 pm By:
FDA Approves Therapy To Treat Gaucher Disease http://retwt.me/1MjGN (via @healthytweet)
Scientists shed light on potential treatment for Gaucher's disease
sciencedaily.com — “In findings that advance scientists' understanding of a whole class of inherited disorders, a research team has shed light on a mechanism that enables a potential treatment for Gaucher's disease and other lysosomal storage diseases.” View full resource at sciencedaily.com
Most Recently Shared on May 10, 2010 at 3:06 am By:
Scientists shed light on potential treatment for Gaucher's disease: In findings that advance scientists' understan... http://bit.ly/c75LfG
FDA Denies Gaucher's Disease Drug; High Jewish Incidence
medicalnewstoday.com — “Gaucher's disease is the most common genetic disease affecting Ashkenazi Jewish people (Eastern, Central and Northern European ancestry), with a carrier frequency of ten percent. Today however, a” View full resource at medicalnewstoday.com
Most Recently Shared on February 25, 2011 at 8:16 pm By:
FDA Denies Gaucher's Disease Drug; High Jewish Incidence http://mnt.to/3RDx #healthnews
Cheaper, More Effective Treatment of Type 1 Gaucher Disease Possible
opa.yale.edu — “Cheaper, More Effective Treatment of Type 1 Gaucher Disease Possible” View full resource at opa.yale.edu
Most Recently Shared on October 20, 2010 at 4:21 pm By:
Elelyso Approved for Type 1 Gaucher Disease --Doctors Lounge
doctorslounge.com — “Elelyso Approved for Type 1 Gaucher Disease --Doctors Lounge” View full resource at doctorslounge.com
Most Recently Shared on May 2, 2012 at 12:04 am By:
Elelyso Approved for Type 1 Gaucher Disease http://t.co/s2NSsCih #Health #Medicine
Healthy Living: Gaucher's disease - YNN, Your News Now
rochester.ynn.com — “Healthy Living: Gaucher's disease - YNN, Your News Now” View full resource at rochester.ynn.com
Most Recently Shared on September 5, 2011 at 9:24 pm By:
Healthy Living: Gaucher's disease: She was supposed to be their star, but now she's battling disease. In today's... http://t.co/2t3lrMZ
Phase 2 Trial Update of Eliglustat for the Treatment of Gaucher Disease - MPR
empr.com — “Genzyme announced four-year follow-up data from its Phase 2 clinical trial of eliglustat tartrate for the treatment of Gaucher disease type 1.” View full resource at empr.com
Most Recently Shared on February 8, 2012 at 8:35 pm By:
Phase 2 Trial Update of Eliglustat for the Treatment of Gaucher Disease http://t.co/cYnJCTvf #hematology
Cheaper, more effective treatment of type 1 Gaucher disease possible
labspaces.net — “Researchers at Yale School of Medicine have found that new disease pathways involving more than one cell type leads to Type 1 Gaucher disease, a rare genetic disorder in which fatty substances called glycosphingolipids accumulate in cells, resulting in liver/spleen enlargement, osteoporosis, bone pain, and increased risk of cancer and Parkinson's disease.” View full resource at labspaces.net
Most Recently Shared on October 20, 2010 at 5:20 pm By:
Cheaper, more effective treatment of type 1 Gaucher disease possible - http://is.gd/g9XvV
Gaucher Disease - Physiopedia - The physiotherapy and physical therapy wiki and encyclopaedia
physio-pedia.com — “Written by physiotherapists and physical therapists for the physiotherapy and physical therapy profession, the aim is to create an international on-line resource for accessible for all rehabilitation and health care professionals.” View full resource at physio-pedia.com
Most Recently Shared on February 28, 2011 at 5:15 pm By:
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