Leber's hereditary optic neuropathy

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

Retrieved on: 
Tuesday, April 9, 2024
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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

Retrieved on: 
Thursday, March 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.

Onconetix Announces Appointment of Seasoned Biotech Executives Dr. Ajit Singh and Dr. Thomas Meier to Its Board of Directors

Retrieved on: 
Tuesday, February 13, 2024
Therapy, School, Sir Ratan Tata Trust, Cadila Pharmaceuticals, Entrepreneurship, AI, Investment, Growth, Silicon, Senior advisor, AACR, Roche, Lead, Partner, Leber's hereditary optic neuropathy, Trustee, Patient, SIX Swiss Exchange, Knowledge, Max Healthcare, Stanford University, Pharmaceutical industry

“We are thrilled to announce the addition of Dr. Singh and Dr. Meier to the Board of Directors of Onconetix,” stated Chairman of the Board James Sapirstein.

Key Points: 
  • “We are thrilled to announce the addition of Dr. Singh and Dr. Meier to the Board of Directors of Onconetix,” stated Chairman of the Board James Sapirstein.
  • Dr. Singh serves as an Adjunct Professor in the School of Medicine at Stanford, where he teaches clinical diagnostics and entrepreneurship.
  • Dr. Singh has held several board positions including Lead Director at Max Healthcare, board member at Cadila Pharmaceuticals, and Senior Advisor to the Tata Trusts Cancer program.
  • He is co-founder, board member and advisor for several biotech companies in Switzerland and the US.

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

Retrieved on: 
Wednesday, January 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.

Coming Soon to a Clinic Near You: Top 12 Emerging Technologies Likely to Impact Patient Care Announced in Annual “Disruptive Dozen” from Mass General Brigham

Retrieved on: 
Wednesday, June 14, 2023
Weight, Smoker, Urinary tract infection, Macular degeneration, Public, Harvard University, Genetic code, Patient, Risk, Menopause, Mass General Brigham, Health, ALS, Disease, Tissue, Heart, CT, Pancreas, Research, Brain, Organization, Mass, Biology, Knowledge, Growth, Diabetic retinopathy, Death, Retina, LLM Lettering, Neuron, DNA, Insulin, Non-Smokers' Rights Association, Biotechnology, COVID-19, Cancer, Lung cancer, Medicine, Paratriathlon, Cell, History, Antibody, Hot, Messenger, Cas9, Translation, Bank of America, RSV, Traction, Immune system, Hunger, Forum, Glaucoma, Radiation, Obesity, Weight loss, Intelligence, Woman, Leber's hereditary optic neuropathy, Inflammation, ATTR, Physician, Conditional sentence, AI, Bank, Pharmaceutical industry, Birth control, Vaccine, Medical imaging

Boston, MA, June 14, 2023 (GLOBE NEWSWIRE) -- Mass General Brigham today revealed the annual “Disruptive Dozen,” 12 emerging technologies with the greatest potential to impact health care in the next several years.

Key Points: 
  • Boston, MA, June 14, 2023 (GLOBE NEWSWIRE) -- Mass General Brigham today revealed the annual “Disruptive Dozen,” 12 emerging technologies with the greatest potential to impact health care in the next several years.
  • The “Disruptive Dozen” results from interviews of Mass General Brigham senior Harvard faculty and a selection process to identify the 12 most likely to have significant impact on patient care in the next few years.
  • The 2023 Mass General Brigham Disruptive Dozen are:
    New drugs that mimic a hormone that curbs hunger, called GLP-1, are blazing a new trail in weight loss.
  • After years of painstaking work, a vaccine that protects against the respiratory syncytial virus (RSV) is at last within reach.

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

Retrieved on: 
Thursday, April 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 Publication of LUMEVOQ® Safety Data over 5-Year Period in renowned Peer-Reviewed American Journal of Ophthalmology

Retrieved on: 
Thursday, March 9, 2023
Research, Neurology, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Optical, Science, MD, Nature, Emergency, Intravitreal injection, American Journal of Ophthalmology, AE, American Journal, Patient, Eye, ISIN, Safety, Leber's hereditary optic neuropathy, Pharmaceutical industry, Euronext, Ophthalmology

It confirms the good overall safety profile of the product in terms of systemic and ocular tolerability, humoral and cellular immune response and highlights a comparable safety profile for unilaterally and bilaterally treated patients.

Key Points: 
  • It confirms the good overall safety profile of the product in terms of systemic and ocular tolerability, humoral and cellular immune response and highlights a comparable safety profile for unilaterally and bilaterally treated patients.
  • "We are very pleased to see our work published in such a prestigious journal, which highlights the significance of our findings.
  • This safety profile was consistent across patients, whether treated bilaterally or unilaterally, in terms of AE, bio-dissemination and immunogenicity.
  • No significant differences in the frequency, nature or severity of systemic and ocular AE were found between these two groups of patients.

Santhera Concludes Agreement with French Authorities on Raxone® Reimbursement and Plans to Submit a Request for an Early Access Program for Vamorolone

Retrieved on: 
Wednesday, February 8, 2023
MAA, File, LHON, Leber's hereditary optic neuropathy, Duchenne muscular dystrophy, European Medicines Agency, NDA, AAP, Patient, FDA, Vamorolone, DMD, EMA, ATU, Health insurance, Pharmaceutical industry, EU, Idebenone

In addition, the Company plans to submit a request in France in the near-term for an early access program for vamorolone for the treatment of Duchenne muscular dystrophy (DMD).

Key Points: 
  • In addition, the Company plans to submit a request in France in the near-term for an early access program for vamorolone for the treatment of Duchenne muscular dystrophy (DMD).
  • Shortly, Raxone will be on the list of reimbursed products in France, and Santhera can resume generating sales.
  • “We are glad to have reached an agreement with the French authorities on the matter of Raxone reimbursement.
  • Earlier in January, Santhera announced FDA acceptance of the new drug application (NDA) for vamorolone for DMD for filing.

Neurophth Receives IND Clearance from FDA for AAV-ND1 Gene Therapy of LHON

Retrieved on: 
Monday, December 19, 2022
Retinal ganglion cell, Phase, NADH, Physician, ND1, EMA, Mitochondrion, Clinical trial, Reactive oxygen species, SAN, LHON, Prevalence, Peripheral neuropathy, Regulatory affairs, Food, FDA, DNA, Axon, ODD, Safety, ND6, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Degenerative disease, Retina, Histidine, EBioMedicine, Cytomegalovirus, Scientific Reports, RGC Resources, Multimedia, Leber's hereditary optic neuropathy, ND4, AAV, IND, Mutation, U.S. FDA, Therapy, Senior, Patient, Pharmaceutical industry, Vaccine, Ophthalmology, Nature

Professor Bin Li, Founder, Chairman and CEO of Neurophth, said, "We are thrilled to have received IND clearance for NFS-02, marking our second program to reach clinical development.

Key Points: 
  • Professor Bin Li, Founder, Chairman and CEO of Neurophth, said, "We are thrilled to have received IND clearance for NFS-02, marking our second program to reach clinical development.
  • Based on encouraging preclinical data, we believe that this gene therapy drug candidate holds promise to become a safe and effective treatment for patients."
  • Our most advanced investigational gene therapy drug candidate, NR082 (rAAV2-ND4), in development for the treatment of mtND4-mediated LHON, has been granted orphan drug designation (ODD) by the U.S. FDA and EMA.
  • Recently, our second gene therapy drug candidate NFS-02, has been granted IND clearance from U.S. FDA.

Santhera’s Shareholders Approve all Board Proposals at Today’s Extraordinary General Meeting

Retrieved on: 
Tuesday, November 29, 2022
Doctor of Philosophy, European Medicines Agency, MAA, Social Security, Communication, REDI (Execution Management System), EMA, Patient, Duchenne muscular dystrophy, FDA, US, Extraordinary general meeting, EGM, DMD, NDA, Cystic fibrosis, Hotel manager, Leber's hereditary optic neuropathy, CHF, Vamorolone, LHON, Security (finance), Television, Pharmaceutical industry

Pratteln, Switzerland, November29, 2022 Santhera Pharmaceuticals (SIX: SANN) announces that its shareholders have approved all proposals by the Board of Directors at the Extraordinary General Meeting (EGM) held today at the Companys domicile in Pratteln, Switzerland.

Key Points: 
  • Pratteln, Switzerland, November29, 2022 Santhera Pharmaceuticals (SIX: SANN) announces that its shareholders have approved all proposals by the Board of Directors at the Extraordinary General Meeting (EGM) held today at the Companys domicile in Pratteln, Switzerland.
  • Increase of authorized capital by CHF 100,000.00 (currently CHF 368,606.87) and its extension until November 28, 2024.
  • Approval of total maximum variable compensation of CHF 2,500,000 for the members of the Executive Management (incl.
  • Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision.