Restoring Vision in Patients with Rare Retinal Diseases with Brian Strem Kiora Pharmaceuticals TRANSCRIPT
Download PDFNov 7, 2023
Brian Strem, President and CEO of Kiora Pharmaceuticals, is developing a small molecule drug to treat orphan retinal diseases. Their drug KIO-301 restores vision in patients with inherited retinal disease. By giving retinal ganglion cells the ability to sense light, signals can be sent to the brain to effectively see...
Nov 7, 2023
Brian Strem, President and CEO of Kiora Pharmaceuticals, is developing a small molecule drug to treat orphan retinal diseases. Their drug KIO-301 restores vision in patients with inherited retinal disease. By giving retinal ganglion cells the ability to sense light, signals can be sent to the brain to effectively see...
Developing Recombinant Protein to Treat Ultra-Rare Hematology Disease cTTP with Dr. Björn Mellgård Takeda TRANSCRIPT
Download PDFAug 21, 2023
Dr. Björn Mellgård, VP and Global Program Lead of rare genetics and hematology at Takeda, is passionate about finding a cure for cTTP, congenital thrombotic thrombocytopenic purpura. This ultra-rare disease, caused by an enzyme deficiency, presents in early childhood and results in life-threatening blood clots....
Aug 21, 2023
Dr. Björn Mellgård, VP and Global Program Lead of rare genetics and hematology at Takeda, is passionate about finding a cure for cTTP, congenital thrombotic thrombocytopenic purpura. This ultra-rare disease, caused by an enzyme deficiency, presents in early childhood and results in life-threatening blood clots....
How Genetic Testing and Real-World Evidence Based Research Shed Light on Ultra-Rare Disease FOXG1 with Nasha Fitter FOXG1 Research Foundation TRANSCRIPT
Download PDFJul 7, 2023
Nasha Fitter, CEO and Co-Founder of the FOXG1 Research Foundation and Vice President of Rare and Neurological Diseases at Ciitizen. Nasha describes the characteristics of the ultra-rare disease FOXG1 Syndrome and the global network that has been built to gain a better understanding of this neurological condition. Using...