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Science & Technology

A total of 158 posts are filed under Science & Technology
FDA Grants MeiraGTx Rare Pediatric Disease Designation to Gene Therapy for Eye Disease
Rare Daily Staff The U. S. Food and Drug Administration granted rare pediatric disease designation to MeiraGTx’s gene therapy product… Continue Reading
Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate in Duchenne
Rare Daily Staff Solid Biosciences said that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin… Continue Reading
New Data Show Survival Improvement with Adcetris in Hodgkin Lymphoma Patients
by Mathew Shanley for Rare Disease Report Earlier today, Takeda and Seattle Genetics announced promising results from its Phase 3 ECHELON-1… Continue Reading
CheckRare: FDA Approves Haegarda to Treat HAE
The U.S. Food and Drug Administration today approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous administration to… Continue Reading
Patient-Inspired Research Uncovers New Link to Rare Disorder
Meeting a young patient with Zellweger syndrome, a rare, life-threatening genetic disease, started a scientific investigation that… Continue Reading
The Atlantic: What If (Almost) Every Gene Affects (Almost) Everything?
By Ed Yong (SOURCE) In 1999, a group of scientists scoured the genomes of around 150 pairs of siblings in an attempt to find genes that are… Continue Reading
Dalhousie Researcher Studying Drug to Combat Fabry Disease
A simple pill that switches off a genetic trigger could potentially provide a powerful treatment for Fabry disease, a rare disorder that… Continue Reading