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Passage Bio Presents New Data for Patients with GM1 Gangliosidosis in Imagine-1 Study

Passage Bio Presents New Data for Patients with GM1 Gangliosidosis in Imagine-1 Study
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Porto alegreRoberto giuglianiBruce goldsmithEliseo salinasGenetics serviceEuropean commissionDrug administrationUpenn gene therapy programDepartment of geneticsMedical genetics serviceGene therapy programPassage bioOrphan drugRare pediatric disease

Paradigm BioPharma welcomes presentation by medical investigators on PPS as a treatment for unmet MPS VI needs

Paradigm Biopharmaceuticals Ltd (ASX:PAR) has drawn attention in the medical community with a presentation from medical investigators introducing the.

United statesSan diegoRoberto giuglianiMichael imperialeParadigm biopharmaceuticals ltdPentosan polysulfate sodiumMaroteaux lamy syndromePrincipal investigatorPolysulfate sodiumOrphanet journal

Passage Bio Presents New Interim Clinical Data for Patients

Both children with late infantile GM1 gangliosidosis in Cohort 1 of the Imagine-1 clinical trial showed developmental improvement in assessments by study.

University of pennsylvaniaUnited statesGwen fisherDavid weinsteinRoberto giuglianiPorto alegreStuart hendersonBruce goldsmithEliseo salinasUniversity of pennsylvania gene therapy programEuropean commissionDrug administrationPassage bio incUpenn gene therapy programExchange commissionGenetics service

REGENXBIO Presents Additional Positive Interim Data from Phase I/II Trial of RGX-121 for the Treatment of MPS II (Hunter Syndrome) at 18th Annual WORLDSymposium™ 2022

/PRNewswire/ RGX-121, a one-time gene therapy for MPS II, continues to be well-tolerated with no drug-related SAEs across three dose levels Dose-dependent.

Roberto giuglianiPorto alegreDana cormackChris brinzeySteve pakolaTechnology platformTechnology platform licenseesDrug administrationExchange commissionCorporate communicationsGenetics serviceSecurities exchangeDepartment of geneticsMucopolysaccharidosis typeHunter syndromeChief medical officer

REGENXBIO Presents Additional Positive Interim Data from Phase I/II Trial of RGX-121 for the Treatment of MPS II (Hunter Syndrome) at 18th Annual WORLDSymposium™ 2022

/PRNewswire/ RGX-121, a one-time gene therapy for MPS II, continues to be well-tolerated with no drug-related SAEs across three dose levels Dose-dependent.

Roberto giuglianiPorto alegreDana cormackChris brinzeySteve pakolaTechnology platformTechnology platform licenseesDrug administrationExchange commissionCorporate communicationsGenetics serviceSecurities exchangeDepartment of geneticsMucopolysaccharidosis typeHunter syndromeChief medical officer

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