Experimental Treatment Earns Breakthrough Therapy Designation for TK2 Deficiency, a Form of Mitochondrial Disease
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Experimental Treatment Earns Breakthrough Therapy Designation for TK2 Deficiency, a Form of Mitochondrial Disease

According to a story from BioSpace, the biopharmaceutical company Modis Therapeutics recently announced that the US Food and Drug Administration (FDA) has granted Breakthough Therapy designation for the company's experimental…

Continue Reading Experimental Treatment Earns Breakthrough Therapy Designation for TK2 Deficiency, a Form of Mitochondrial Disease
67 Year Old Non-Hodgkin’s Lymphoma Patient Given Second Chance at Life After Experimental Cell Therapy
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67 Year Old Non-Hodgkin’s Lymphoma Patient Given Second Chance at Life After Experimental Cell Therapy

  A grandmother of four in Maryland has recently become the first cancer patient to complete the experimental CAR-T cell procedure at Georgetown University Hospital. Daisy Diggs 67, who was…

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Experimental Treatment for Macular Telangiectasia Type 2 Earns Fast Track Designation

According to a story from prnewswire.com, the biopharmaceutical company Neurotech Pharmaceuticals recently announced that the company had earned Fast Track designation from the US Food and Drug Administration (FDA). This…

Continue Reading Experimental Treatment for Macular Telangiectasia Type 2 Earns Fast Track Designation

Study Hopes to Improve Diagnosis of Niemann-Pick Disease and Other Rare Conditions

The first step in treating rare diseases is accurately diagnosing them. Unfortunately far too many people go years without knowing what is going wrong in their body. Doctors are trained…

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Choroideremia Can Lead to Blindness – But Gene Therapy Might Be Able to Halt It
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Choroideremia Can Lead to Blindness – But Gene Therapy Might Be Able to Halt It

An article recently published by the Guardian shared the stories of three families affected by recent breakthroughs in gene therapy treatments. What is Gene Therapy? Gene therapy is an increasingly…

Continue Reading Choroideremia Can Lead to Blindness – But Gene Therapy Might Be Able to Halt It
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