LHON

Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Positive

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火曜日, 4月 9, 2024
Eurostat, Diagnosis, Civil service commission, Leber's hereditary optic neuropathy, Prevalence, Cell, Patient, Committee, Knowledge, Seriousness, Mitochondrion, Regulation, EMA, IRIS, Adolescence, LHON, European, COMP, Oxygen, Safety, European Commission, Marketing, Visual impairment, Medicine, Pharmaceutical industry

Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Positive

Key Points: 


Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Positive

Team Panasonic Gears Up for the Olympic and Paralympic Games Paris 2024 with Addition of Paralympian Noah Malone to Its Roster of Inspirational Athletes

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木曜日, 3月 28, 2024
Panasonic, Leber's hereditary optic neuropathy, T12, Gold, Branding, University, Indiana State University, Athlete, Life, LHON, World championship, Disability, TED (conference), DSM-IV codes, Cassette tape

NEWARK, N.J., March 28, 2024 /PRNewswire/ -- Today, Panasonic welcomes U.S. Paralympic gold and silver medalist and World Champion in the 100-meter sprint, Noah Malone, to Team Panasonic, bolstering its roster of North American Olympians and Paralympians leading up to the Olympic and Paralympic Games Paris 2024. 

Key Points: 
  • NEWARK, N.J., March 28, 2024 /PRNewswire/ -- Today, Panasonic welcomes U.S. Paralympic gold and silver medalist and World Champion in the 100-meter sprint, Noah Malone, to Team Panasonic, bolstering its roster of North American Olympians and Paralympians leading up to the Olympic and Paralympic Games Paris 2024.
  • Together, this team of athletes will work to raise awareness of Panasonic's social good initiatives.
  • Panasonic has a long history as a proud partner of the Olympic and Paralympic Games.
  • As the exclusive provider of audio-video equipment, Panasonic and its technology has helped to power the Games for over 30 years.

GenSight Biologics Announces Update on Real-World Data from Early Access Programs of LUMEVOQ® Gene Therapy at NANOS 2024

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水曜日, 3月 6, 2024
Health, Neurology, Genetics, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, AAC, Doctor of Philosophy, Multimedia, MD, Inflammation, Mendelian traits in humans, EAPS, Visual acuity, Safety, Q3, University, LHON, ND4, Patient, EAP

In both groups, the clinical improvement exceeded the conventional definition of clinical meaningfulness, in which a 15-letter gain is considered meaningful.

Key Points: 
  • In both groups, the clinical improvement exceeded the conventional definition of clinical meaningfulness, in which a 15-letter gain is considered meaningful.
  • The safety profile of the gene therapy continues to be favorable and comparable between bilaterally treated patients and unilaterally treated patients.
  • “Witnessing results like these energizes the GenSight team’s drive to restore early access availability for eligible patients by Q3 this year,” said Laurence Rodriguez, Chief Executive Officer of GenSight.
  • The Company aims to resume early access in France under the Autorisation de l’accès compassionnel (AAC) program in Q3 2024.

GenSight Biologics Announces LUMEVOQ® Scientific Updates at NANOS 2024

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月曜日, 3月 4, 2024
Oncology, Health, Other Health, Clinical Trials, Pharmaceutical, Optical, Biotechnology, Master of Science, Quality of life, Hospital, University of Cambridge, MT-ND4, University, LHON, Patient

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that new scientific data and analyses on the gene therapy LUMEVOQ® will be presented at the 50th Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS) in Honolulu, Hawaii (March 2-7, 2024).

Key Points: 
  • GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that new scientific data and analyses on the gene therapy LUMEVOQ® will be presented at the 50th Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS) in Honolulu, Hawaii (March 2-7, 2024).
  • Leading Leber Hereditary Optic Neuropathy (LHON) Key Opinion Leaders will provide updates on evidence related to the contralateral effect, on real-world experience with LUMEVOQ®, and on the comparison between LHON natural history, idebenone and LUMEVOQ®.
  • Poster presentation: “Lenadogene nolparvovec gene therapy improves visual recovery more than idebenone in m.11778G>A MT-ND4 LHON, and both treatments exceed the natural course of the disease”
    Presenter: Nancy J. Newman, MD, Emory University School of Medicine, Atlanta, Georgia, USA
    In addition, Benson S. Chen, MBChB, MSc, FRACP, Department of Clinical Neurosciences, University of Cambridge and Cambridge Eye Unit, Addenbrooke’s Hospital, United Kingdom, will present evidence on the quality of life of LHON patients in a platform presentation.
  • Platform presentation: “Vision-Related Quality of Life in LHON Patients Treated with Lenadogene Nolparvovec Gene Therapy: Analysis of the VFQ-25 Using Rasch Measurement Theory”

NAYA Biosciences To Acquire Clinical Stage Gene Therapy Program for Leber's Hereditary Optic Neuropathy (LHON)

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火曜日, 1月 23, 2024
Patient, Regenerative medicine, Fertility, Paratriathlon, AAV, Intercept Pharmaceuticals, Kite Pharma, University, Mitochondrion, Term sheet, LHON, Shearson, Acquisition, Safety, Vice, Intellectual property, FDA, Capsid, Letter, Arsenic trioxide, Paramount, Pharmaceutical industry

“The NAYA leadership team brings an agile entrepreneurial platform, broad development and commercialization experience, and access to public capital, which will unlock the potential of our promising AAV gene therapy platform for mitochondrial genetic diseases.

Key Points: 
  • “The NAYA leadership team brings an agile entrepreneurial platform, broad development and commercialization experience, and access to public capital, which will unlock the potential of our promising AAV gene therapy platform for mitochondrial genetic diseases.
  • The program has received over $6 million in grant funding to date and qualifies for Regenerative Medicine Advanced Therapy (RMAT) designation and multiple priority FDA review vouchers.
  • The combination of multiple orphan indications, multiple routes of administration, and multiple AAV serotypes applicable supports a broad gene therapy platform for mitochondrial orphan diseases.
  • The Florida Biotechnologies Transaction is contingent on the closing of the INVO Merger and sufficient financing to further develop the gene therapy programs from Florida Biotechnologies.

Bringing Ophthalmology Trials into Focus: Reducing Patient Burden, Improving Efficiency and Incorporating Cell and Gene Therapy, Upcoming Webinar Hosted by Xtalks

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水曜日, 1月 10, 2024
Eye, Patient, Therapy, RAPT, CPM, Data collection, LHON, Quality of life, PMP, Caregiver, Advocate, Child, Doctor of Philosophy, Leber's hereditary optic neuropathy, Pharmaceutical industry

TORONTO, Jan. 10, 2024 /PRNewswire-PRWeb/ -- Discover an informative webinar exploring patient-centered approaches shaping ophthalmology trials, uniting stakeholders for better treatments and outcomes. Clinical trials in ophthalmology require special considerations at every stage, including trial design, recruitment strategies, patient outreach and engagement, assessments and data collection and management of investigational products or devices.

Key Points: 
  • In this free webinar, learn about patient-centered approaches shaping ophthalmology trials, uniting stakeholders for better treatments and outcomes.
  • TORONTO, Jan. 10, 2024 /PRNewswire-PRWeb/ -- Discover an informative webinar exploring patient-centered approaches shaping ophthalmology trials, uniting stakeholders for better treatments and outcomes.
  • Clinical trials in ophthalmology require special considerations at every stage, including trial design, recruitment strategies, patient outreach and engagement, assessments and data collection and management of investigational products or devices.
  • The key to success is putting the patients and their caregivers at the center when considering the overall approach to the trial.

Santhera Announces Half-Year 2023 Financial Results and Provides Corporate Update

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木曜日, 9月 7, 2023
Vamorolone, Marketing, European Medicines Agency, Lead, Prescription Drug User Fee Act, Committee, U.S. FDA, Patient, Committee for Medicinal Products for Human Use, News, Duchenne muscular dystrophy, Glucocorticoid, Becker muscular dystrophy, European Commission, EMA, CEST, PDUFA, Rare, FDA, Safety, Health care, LR, Duchenne, Publication, MAA, MHRA, CHMP, Catalyst Pharmaceuticals, Partnership, European Directive on Traditional Herbal Medicinal Products, NDA, BMD, EC, BST, Medical device, Pharmaceutical industry, Security (finance), Idebenone, EU, Chiesi Farmaceutici, LHON, Atlantic Ocean, Catalysis, DMD

Furthermore, we have secured a solid financial footing which allows us to press ahead toward our goal of European commercialization,” said Dario Eklund, CEO of Santhera.

Key Points: 
  • Furthermore, we have secured a solid financial footing which allows us to press ahead toward our goal of European commercialization,” said Dario Eklund, CEO of Santhera.
  • Total consideration to Santhera is up to USD 231 million (including equity investment) plus royalty payments from product sales.
  • After closing of the transaction in July 2023, Santhera received an upfront payment of USD 90 million (USD 75 million in cash and USD 15 million equity investment).
  • During the six months ending June 30, 2023, Santhera committed to the sale of Raxone/idebenone upon settlement of the French case and the transaction closed on July 28, 2023.

Santhera Completes Divestment of Raxone®/Idebenone Business to Chiesi Group

Retrieved on: 
月曜日, 7月 31, 2023
Vamorolone, Marketing, LR, USD, EUR, Patient, FDA, Pharmaceutical industry, Chiesi Farmaceutici, Idebenone, LHON

53 LR

Key Points: 
  • 53 LR
    Santhera retains contingent value for LHON in U.S. and/or other indications worldwide
    Pratteln, Switzerland, July 31, 2023 – Santhera Pharmaceuticals (SIX: SANN) announces the full divestment of its Raxone®/idebenone business worldwide and for all indications to Chiesi Farmaceutici S.p.A., an international research focused healthcare group (Chiesi Group).
  • Under the terms of the agreement, Chiesi Group acquired all assets and certain liabilities related to idebenone in all indications worldwide, including Raxone in LHON, for which Chiesi already held exclusive license rights globally since 2019, except for North America and France.
  • The agreement simplifies the Raxone business significantly for both companies with Chiesi becoming the global brand owner while enabling Santhera to focus on the launch of vamorolone in Europe, subject to approval.
  • Chiesi Group also had the option to fully acquire said business outside North America.

Santhera Announces Preliminary Unaudited 2022 Annual Results Ahead of Full Report Publication by End of May and Provides Corporate Update

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木曜日, 4月 27, 2023
Vamorolone, Prescription Drug User Fee Act, Duchenne muscular dystrophy, Civil service commission, Education, Vitamin D deficiency, Health care, Glucocorticoid, European Medicines Agency, NDA, LR, European Directive on Traditional Herbal Medicinal Products, Biomarker, BMD, Committee, EMA, Vomiting, Depression, Prednisone, Publication, DMD, Vertebral compression fracture, PAMS, Patient, Annual report, Leber's hereditary optic neuropathy, European Commission, CEST, Syndrome, PDUFA, Committee for Medicinal Products for Human Use, MAA, FDA, LHON, CHF, EC, Safety, News, Bone fracture, MHRA, CHMP, BST, Cryptocurrency, Pharmaceutical industry, Medical device, Dietary supplement, Dentistry, EU, Idebenone

As permitted by SIX Exchange Regulation, the Company will publish the full 2022 Annual Report by the end of May.

Key Points: 
  • As permitted by SIX Exchange Regulation, the Company will publish the full 2022 Annual Report by the end of May.
  • The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency.
  • SIX Exchange Regulation has permitted Santhera to publish its 2022 Annual Report by May 31, 2023, at the latest.
  • The Company plans to publish its audited 2022 Annual Report during May 2023.

GenSight Biologics Announces Presentation of LUMEVOQ® Efficacy and Safety Data from Early Access Programs for ND4-LHON Patients at NANOS 2023

Retrieved on: 
水曜日, 3月 15, 2023
Health, Genetics, Research, Science, Pharmaceutical, Optical, Biotechnology, Multimedia, Injection, EAP, Eye, MD, EAPS, Hospital, Neuro-ophthalmology, LHON, ISIN, Patient, Annual Meetings of the International Monetary Fund and the World Bank Group, ND4, Safety, Intravitreal injection, Pharmaceutical industry, Medical imaging, Mendelian traits in humans, Ophthalmology, Euronext

View the full release here: https://www.businesswire.com/news/home/20230314006021/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20230314006021/en/
    Figure 1: Global evolution of mean BCVA over two years in patients who received LUMEVOQ® in early access programs.
  • Individual data from 45 out of the 63 patients who had passed the one-year post-treatment point and performed their one-year visit were pooled and analyzed1.
  • The data confirmed the efficacy and safety profile of LUMEVOQ® in a real-world setting.
  • Figure 1: Global evolution of mean BCVA over two years in patients who received LUMEVOQ® in early access programs.