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Frontiers | Exploiting hiPSCs in Leber's Hereditary Optic Neuropathy (LHON): Present Achievements and Future Perspectives

More than thirty years after discovering Leber’s hereditary optic neuropathy (LHON) as the first maternally inherited disease associated with homoplasmic mtDNA mutations, we still struggle to achieve effective therapies. LHON is characterized by selective degeneration of retinal ganglion cells (RGCs) and is the most frequent mitochondrial disease, which leads to blindness young people, in particular males. Despite the causative mutations are present in all tissues, yet only a specific cell type is affected. Our deep understanding of the pathogenic mechanisms in LHON is hampered by the lack of appropriate models since investigations have been traditionally performed in non-neuronal cells. Effective in vitro models of LHON are now emerging, casting promise to speed our understanding of pathophysiology and test therapeutic strategies to accelerate translation into clinic. We here review the potentials of these new models and their impact on the future of LHON patients. ....

South Korea , Trentino Alto Adige , Friuli Venezia Giulia , Ch Ungch Ong Namdo , Doug Wallace , Istituto Neurologico Carlo Besta , Aderm Organoid , Neurogastrointestinal Encephalomyopathy , Izpisua Belmonte , Artero Castro , Carelliv Leber , Acta Ophthalmol , Biophysica Acta , Associazione Luigi Comini , European Reference Network For Rare Neuromuscular Diseases , Italian Ministry Of Health , Mariani Foundation , Research Topic , Drug Administration , European Medicines Agency , Peripheral Blood Mononuclear Cells , Neurons From , Adeno Associated Virus , Neurologico Carlo , Italian Ministry , Mitochondrial Pediatric Diseases ,

GW Pharmaceuticals receives European Commission approval for EPIDYOLEX® (cannabidiol) for the ...


Press release content from Globe Newswire. The AP news staff was not involved in its creation.
GW Pharmaceuticals receives European Commission approval for EPIDYOLEX® (cannabidiol) for the .
GW Pharmaceuticals plcApril 20, 2021 GMT
– Tuberous sclerosis complex (TSC) represents a third indication for GW’s cannabidiol in Europe –
– TSC causes epilepsy in up to 85% of patients, and up to 60% of those patients do not respond to standard anti-epileptic medicines
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LONDON, April 20, 2021 (GLOBE NEWSWIRE) GW Pharmaceuticals plc (Nasdaq: GWPH) (“GW”, “the Company” or “the Group”), a world leader in discovering, developing and delivering regulatory approved cannabis-based medicines, today announces that the European Commission (EC) has approved the Type II variation application for EPIDYOLEX® (cannabidiol) as an adjunctive treatment of seizures associated with TSC, for patients two years of age and older. This approval, which represen ....

United Kingdom , City Of , Volker Knappertz , Alexis Arzimanoglou , Ben Atwell , Kristen Cardillo , Scott Giacobello , Globe Newswire Gw Pharmaceuticals , European Commission , Drug Administration , Gw Pharmaceuticals , European Union , Epicare University Hospitals Of Lyon , Corporate Communications , European Medicines Agency , Justin Gover , Medical Officer , European Reference Network , Complex Epilepsies , University Hospitals , Lennox Gastaut Syndrome , Greenwich Biosciences , United Kingdom Based , Tuberous Sclerosis Complex , Orphan Drug Designation , Sclerosis Complex ,

GW Pharmaceuticals plc: GW Pharmaceuticals receives European Commission approval for EPIDYOLEX (cannabidiol) for the treatment of seizures associated with tuberous sclerosis complex


GW Pharmaceuticals plc: GW Pharmaceuticals receives European Commission approval for EPIDYOLEX (cannabidiol) for the treatment of seizures associated with tuberous sclerosis complex
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LONDON, April 20, 2021 (GLOBE NEWSWIRE) GW Pharmaceuticals plc (Nasdaq: GWPH) ( GW , the Company or the Group ), a world leader in discovering, developing and delivering regulatory approved cannabis-based medicines, today announces that the European Commission (EC) has approved the Type II variation application for EPIDYOLEX (cannabidiol) as an adjunctive treatment of seizures associated with TSC, for patients two years of age and older. This approval, which represents the third indication for GW s cannabidiol in Europe, paves the way for the expanded launch of the medicine across Europe. ....

United States , United Kingdom , City Of , Volker Knappertz , Alexis Arzimanoglou , Ben Atwell , Kristen Cardillo , Scott Giacobello , Ts Alliance Website , Globe Newswire Gw Pharmaceuticals , European Commission , Drug Administration , Gw Pharmaceuticals , European Union , Epicare University Hospitals Of Lyon , Corporate Communications , European Medicines Agency , Justin Gover , Medical Officer , European Reference Network , Complex Epilepsies , University Hospitals , Lennox Gastaut Syndrome , Tuberous Sclerosis Complex , Orphan Drug Designation , Sclerosis Complex ,