Latest Breaking News On - Rare pediatric disease - Page 16 : comparemela.com

REGENXBIO Announces Pivotal Trial of RGX-121 for the Treatment of MPS II Achieves Primary Endpoint

REGENXBIO Announces Pivotal Trial of RGX-121 for the Treatment of MPS II Achieves Primary Endpoint
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Paul harmatzChris brinzeyDana cormackRaymond wangEuropean medicines agencyTechnology platformSecurities exchangeTechnology platform licenseesCorporate communicationsDrug administrationExchange commissionMucopolysaccharidosis typeRare pediatric disease priority review voucherOrange countyBiologics license applicationOrphan drug product

Aro Biotherapeutics to Present Updates on ABX1100, a CD71 Centyrin ? Gys1 siRNA Conjugate for the Treatment of Pompe Disease at the 20th Annual WORLD Symposium 2024? Meeting

Aro Biotherapeutics to Present Updates on ABX1100, a CD71 Centyrin ? Gys1 siRNA Conjugate for the Treatment of Pompe Disease at the 20th Annual WORLD Symposium 2024? Meeting
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United statesSan diegoSteven nadlerBartholomew pedersonTranslational research at aro biotherapeuticsDrug administrationTranslation research of aro biotherapeuticsProduct servicePresent updatesPompe diseaseMedical educationBall state universityLysosomal storage diseaseSenior vice presidentTranslation researchOrphan drug designation

Takeda and Protagonist Therapeutics, Inc Enter into Worldwide License and Collaboration Agreement for Rusfertide, a Late-Stage Rare Hematology Asset

Takeda and Protagonist Therapeutics, Inc Enter into Worldwide License and Collaboration Agreement for Rusfertide, a Late-Stage Rare Hematology Asset
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United kingdomUnited statesDineshv patelAnn hematolJulie kimUs coJapan ministry of healthEuropean medicines agencyTakeda pharmaceutical company limitedDrug administrationTakeda pharmaceuticals united statesa incExchange commissionProtagonist therapeutics incAmerican society of hematologyBe protagonist coTakeda will be protagonist

Takeda and Protagonist Therapeutics, Inc Enter into Worldwide License and Collaboration Agreement f

Takeda Will Be Protagonist's Co-Development, U.S. Co-Commercialization Partner With 50:50 Profit Share, and With Exclusive Ex-U.S. Global Rights to Commercialize Rusfertide, Protagonist's Investigational Injectable Hepcidin Mimetic Currently in Development for the Treatment of Polycythemia Vera (PV)Takeda Will Make.

United statesUnited kingdomAnn hematolDineshv patelJulie kimBe protagonist coTakeda pharmaceutical company limitedDrug administrationExchange commissionTakeda pharmaceuticals united statesa incEuropean medicines agencyProtagonist therapeutics incAmerican society of hematologyJapan ministry of healthUs coTakeda will be protagonist

Tyra Biosciences Receives FDA Rare Pediatric Disease Designation for TYRA-300 for the Treatment of Achondroplasia

Tyra Biosciences Receives FDA Rare Pediatric Disease Designation for TYRA-300 for the Treatment of Achondroplasia
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United statesHiroomi tadaAmy conradTyra biosciencesPrnewswire tyra biosciences incTyra biosciences incExchange commissionDrug administrationFibroblast growth factor receptorRare pediatric diseaseInvestigational new drugRare pediatric disease designationChief medical officerPediatric diseaseNew drug applicationPriority review voucher