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Research

A total of 248 posts are filed under Research
New Study Offers Hope for Patients Suffering from a Rare Form of Blindness
Rare Daily Staff A new form of therapy may halt or even reverse a form of progressive vision loss that, until now, has inevitably led to… Continue Reading
Study Shows Cholic Acid Replacement Leads to Favorable Outcome in Adulthood for Children with Rare Genetic Liver Diseases
Rare Daily Staff While oral cholic acid replacement therapy has been shown to be an effective treatment for children with primary bile acid… Continue Reading
Experimental Drug More Effective in Treating Rare Kidney Disease
Rare Daily Staff A new phase 2 study found that patients with the rare disease focal segmental glomerulosclerosis (FSGS) taking the… Continue Reading
Amicus Forms R&D Collaboration with Penn Focused on AAV Gene Therapies
Rare Daily Staff Amicus Therapeutics said it entered into a collaboration with the Gene Therapy Program in the Perelman School of… Continue Reading
Discovery Could Lead to Better Diagnosis and Treatment of Lupus in African Patients
Rare Daily Staff Black Africans have a higher level of Lupus than previous known and identified an antibody previously unlinked to the… Continue Reading
NIH Expands Undiagnosed Diseases Network with $100 Million in New Grants
Rare Daily Staff The National Institutes of Health said it would provide about $100 million in grants over the next four years to expand its… Continue Reading
Thinking Ahead
When the European Medicines Agency approved Myozyme in 2006, Genzyme’s enzyme replacement therapy for the lysosomal storage disorder… Continue Reading
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