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Genetics

A total of 24 posts are filed under Genetics
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India Raring to Make its Mark in the Rare Disease Market
With one-third of all new approved drugs over the past five years in the US addressing rare diseases, the last decade is said to have been… Continue Reading
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New University of Ottawa Shows DMD Impacts Stem Cells
New treatments could one day be available for the most common form of muscular dystrophy after a study suggests the debilitating genetic… Continue Reading
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News Medial: Rice Scientists Solve Mystery About Hemophilia Protein
Rice University scientists have solved a long-standing mystery about where the body stores and deploys blood-clotting factor VIII, a protein… Continue Reading
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FDA Awards KGI an Orphan Drug Designation for Rett Syndrome
A project that was part of Dr. Ian Phillips’ Writing an Orphan Drug Application (ALS 432) class recently led to the U.S. Food and Drug… Continue Reading
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Spark Therapeutics Announces Results from Trial of SPK-RPE65 for Genetic Blinding Conditions
Spark Therapeutics today announced positive results from their Phase 3 pivotal trial for a gene therapy product looking to be used to treat… Continue Reading
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New York Times Features Children With RCDP, a Painful and Fatal Form of Dwarfism
Once a year, Crystal and Jonathan Bedford drive 1,000 miles from their home in Texas to rural Alabama, their three children in tow. Beside a… Continue Reading
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Congenica Welcomes Cambridge Rare Disease Network Initiative
The first sign that a child has a rare disease is often when they fail to reach key developmental milestones or, alternatively, when they… Continue Reading