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A total of 25 posts are filed under Genetics
Stem Cell Gene Therapy Shows Promise In Treating Patients With Rare Immunodeficiency Disorder
According to scientists from the National Institute of Allergy and Infectious Diseases, gene therapy can safely restore the immunity of… Continue Reading
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
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
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
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
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
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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