CHOP Researchers: Patient-Centric Perspective Offers Key Insights into Upcoming Treatment Studies Philadelphia, June 6, 2018 – As clinical trials gear up with the aim of attaining the first FDA-approved treatments for mitochondrial disease, a new study reports for the first time what patients and families say would motivate them for or against participating in [...]
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So far Cliff Gorski has created 71 blog entries.
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Stealth BioTherapeutics Granted Orphan Drug Designation of Elamipretide for Treatment of Leber’s Hereditary Optic NeuropathyCliff Gorski2018-04-30T10:06:05+00:00
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Click here to see the information on this paper. Listen to the Podcast with Dr. Hirano and Dr. Thompson
GenSight has demonstrated that its gene therapy for a rare disease causing blindness can significantly improve the vision of LHON patients.Cliff Gorski2018-04-04T13:09:37+00:00
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Preclinical Testing in 3 Model Systems Suggests Some Antioxidants May Be Effective Mitochondrial Disease TreatmentsCliff Gorski2018-03-27T12:48:54+00:00
Preclinical Testing in 3 Model Systems Suggests Some Antioxidants May Be Effective Mitochondrial Disease Treatments --CHOP Researchers Test 7 Compounds in Seeking Candidates for Clinical Trials; Early Efficacy Seen in NAC and Vitamin E-- Philadelphia, March 27, 2018 – A systematic study of seven antioxidants commonly taken by or suggested to benefit children [...]
See the letter by patient advovcacy groups in opposition here.
Intravenous Arginine Benefits Children after Acute Metabolic Strokes — CHOP Researchers Find Notable Responses, No Adverse Events in Children with Mitochondrial DiseaseCliff Gorski2018-03-09T11:09:56+00:00
FOR IMMEDIATE RELEASE Friday, March 9, 2018 John Ascenzi Children’s Hospital of Philadelphia (267) 426-6055 Ascenzi@email.chop.edu Intravenous Arginine Benefits Children after Acute Metabolic Strokes --CHOP Researchers Find Notable Responses, No Adverse Events in Children with Mitochondrial Disease-- Philadelphia, March 9, 2018 – Children with mitochondrial diseases who suffered acute metabolic strokes benefited from [...]