|Signs and Symptoms||Approximate number of patients (when available)|
(present in 5%-29% of cases)
|Autosomal dominant inheritance||-|
|Autosomal recessive inheritance||-|
|Skeletal muscle atrophy||-|
Research helps us better understand diseases and can lead to advances in diagnosis and treatment. This section provides resources to help you learn about medical research and ways to get involved.
Living with a genetic or rare disease can impact the daily lives of patients and families. These resources can help families navigate various aspects of living with a rare disease.
Third NIH Workshop on Gaucher Disease and Parkinsonism Thursday, April 8, 2010 -
Friday, April 9, 2010
Location: NIH- the Cloister, Bethesda, Maryland
Description: Workshop goals were to (1) determine the appropriate human, animal, or cell-based models to better understand this correlation; (2) explore the mechanisms and/or pathways involved; and (3) establish international collaborations to enhance progress in the field and to make recommendations for future study.
Contact: Dr. Ellen Sidransky301email@example.com
Co-funding Institute(s): National Human Genome Research Institute, Office of Rare Diseases Research
Questions sent to GARD may be posted here if the information could be helpful to others. We remove all identifying information when posting a question to protect your privacy. If you do not want your question posted, please let us know. Submit a new question
If someone has MSA, is it possible that they do things with out realizing what they are doing is wrong? Does this disease have any impact on them being unable to differ right from wrong. I'm asking this because someone I know has been recently diagnosed with MSA and is in the final stage, he has done some pretty unfavorable things in the past and people are trying to justify this disease played a factor in his mental stability at the time. Is this possible? See answer