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    Blue test tubes arranged in a line, disappearing into the background
    Drug news
    29/06/15

    CHMP recommends Raxone (idebenone) to treat Leber's Hereditary Optic Neuropathy (LHON)- Santhera

    The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation for Raxone (idebenone)...

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    Drug news
    13/03/19

    Santhera Pharma completes enrollment in phase IV (LEROS) study of Raxone to treat Leber's hereditary optic neuropathy

    Santhera Pharmaceuticals announces that it has completed patient enrollment in the ongoing Phase IV study (LEROS) with Raxone (idebenone) for...

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    Drug news
    16/09/17

    CHMP adopts negative opinion for its Type II extension application for Raxone (idebenone) in Duchenne muscular dystrophy .- Santhera Pharmaceuticals

    Santhera Pharmaceuticals) announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has...

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    Drug news
    30/03/19

    Santhera update on filing for conditional marketing authorization in Europe for Puldysa (idebenone) in Duchenne Muscular Dystrophy.

    Santhera has expanded and substantiated its previous regulatory dossier with additional clinical data from patients treated with idebenone, new analyses...

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    Drug news
    19/01/13

    EMA rejects Raxone/Catena (Santhera) as a treatment for Leber's Hereditary Optic Neuropathy

    Santhera Pharmaceuticals has announced that it has received a negative opinion on its Marketing Authorization Application (MAA) for Raxone (idebenone)...

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    Drug news
    09/09/15

    EU approves Raxone (idebenone), to treat visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (LHON)- Santhera Pharma

    Santhera Pharmaceuticals announces the European Commission (EC) has granted marketing authorization for Raxone (idebenone) as the first approved medicine available...

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    Drug news
    31/07/23

    Santhera completes divestment of Raxone/Idebenone business to Chiesi Group.

    Santhera Pharmaceuticals 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). The transaction replaces the existing license agreement between the two companies entered into in 2019

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    Drug news
    08/02/22

    REFLECT phase III clinical trial for Lumevoq shows benefits for bilateral treatment in Leber Hereditary Optic Neuropathy.- GenSight Biologics

    GenSight Biologics reported key efficacy and safety findings at 1.5 years (78 weeks) post-treatment in the REFLECT Phase III clinical trial for Lumevoq. The results show better visual acuity improvements from bilateral intravitreal injections of the gene therapy compared to a unilateral injection. The REFLECT trial is a randomized, double-masked, placebo-controlled Phase III trial involving 98 subjects with vision loss due to Leber Hereditary Optic Neuropathy (LHON) caused by a mutated ND4 mitochondrial gene; enrolled ND4 subjects had vision loss up to one year from onset.

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    Drug news
    25/01/18

    Negative opinion for Raxone (idebenone) in Duchenne muscular dystrophy expected.

    Santhera Pharmaceuticals announced oral explanation at the CHMP of the EMA.

    Journals
    Journal
    01/03/23

    Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial

    Background: The study objective was to assess the effect of vutrisiran, an RNA interference therapeutic that reduces transthyretin (TTR) production, in patients with hereditary transthyretin (ATTRv) amyloidosis with polyneuropathy.

    • Source: Amyloid
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