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    Clinical trial
    08/06/15

    Safety and Efficacy of a Switch to MK-1439A in Human Immunodeficiency Virus (HIV-1)-Infected Participants Virologically Suppressed on an Anti-retroviral Regimen in Combination With Two Nucleoside Reverse Transcriptase Inhibitors (MK-1439A-024) (DRIVE-SHIF

    The multicenter, open label, randomized study will evaluate the safety and efficacy of a switch to MK-1439A (MK-1439 [doravirine] plus lamivudine and tenofovir disoproxil fumarate) in HIV-1-infected participants virologically suppressed...

    • Source: clinicaltrials.gov
    Guidelines
    Guideline
    10/06/22

    European Headache Federation guideline on the use of monoclonal antibodies targeting the calcitonin gene related peptide pathway for migraine prevention – 2022 update

    This 2022 EHF guideline update reviews the use of monoclonal antibodies targeting the CGRP pathway for migraine prevention. It incorporates new RCTs and real-world data to support the efficacy and safety of eptinezumab, erenumab, fremanezumab, and galcanezumab in both episodic and chronic migraine, with expert guidance on long-term management.

    • Source: European Headache Federation
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    Clinical trial
    07/10/24

    Gene transfer study in patients with late onset Pompe disease (FORTIS)

    This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).

    • Source: ClinicalTrials.gov
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    Clinical trial
    02/05/24

    Open-label, Pivotal Clinical Trial to Confirm Efficacy and Safety of Autologous Grafts Containing Stem Cells Genetically Modified for Epidermis Restoration in Patients With Junctional Epidermolysis Bullosa (HOLOGENE 5)

    Prospective, multicenter and multinational, open-label, uncontrolled clinical study to assess the safety and efficacy of autologous cultured epidermal grafts containing epidermal stem cells genetically modified transduced with a LAMB3-gamma retroviral vector.

    • Source: ClinicalTrials.gov
    Journals
    Journal
    31/05/18

    Eltrombopag in immune thrombocytopenia: efficacy review and update on drug safety.

    This review summarizes the experience reported using eltrombopag in ITP, paying attention to efficacy and safety.

    • Source: Therapeutic Advances in Drug Safety
    Journals
    Journal
    01/02/21

    Gene therapy for neovascular age-related macular degeneration: rationale, clinical trials and future directions

    In this review we aim to provide an update of the current status of gene therapy for nAMD and briefly discuss future prospects.

    • Source: British Journal of Ophthalmology
    Journals
    Journal
    01/03/20

    The role of hypoxia-inducible factors in neovascular age-related macular degeneration: a gene therapy perspective

    The present review provides an update on current gene therapies in ocular angiogenesis, particularly nAMD, from both basic and clinical perspectives.

    • Source: Cellular and Molecular Life Sciences
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    Clinical trial
    07/10/24

    Evaluation of the safety and efficacy of infantile-onset Pompe Disease gene therapy drug

    This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease.

    • Source: ClinicalTrials.gov
    Journals
    Journal
    31/01/19

    Ixekizumab for the treatment of psoriasis: an update on new data since first approval

    Areas covered: In this review, we summarize the latest clinical study results on ixekizumab. Long-term Phase III study data on efficacy and safety are now available for both plaque psoriasis and psoriatic arthritis.

    • Source: Expert Review of Clinical Immunology
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    Clinical trial
    15/12/24

    Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene

    This Phase I/II study explores the safety and efficacy of a novel gene therapy for retinitis pigmentosa (RP) caused by PDE6B mutations. By delivering the therapeutic PDE6B gene via subretinal administration of AAV2/5-hPDE6B, the study aims to restore retinal function in affected patients.

    • Source: Clinicaltrials.gov
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