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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
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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
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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
    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
    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
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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
    Journals
    Journal
    01/06/21

    Nephropathic cystinosis: an update on genetic conditioning

    Discover the complexities of cystinosis, a genetic disorder caused by CTNS gene mutations, leading to toxic cystine crystal accumulation in lysosomes. This review explores the geographic variations in cystinosis, its clinical manifestations, and the need for novel treatments beyond cysteamine therapy.

    • Source: Pediatric Nephrology
    A close up image of a microscope
    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
    31/05/18

    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
    01/04/22

    In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial

    Recessive dystrophic epidermolysis bullosa (RDEB) is a lifelong genodermatosis associated with blistering, wounding, and scarring caused by mutations in COL7A1, the gene encoding the anchoring fibril component, collagen VII (C7).

    • Source: Nature Medicine
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