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Ultragenyx Receives FDA Agreement to Expand Ongoing Global Phase 1/2 Trial Evaluating GTX-102 in Patients with Angelman Syndrome in the U S

Amended U.S. protocol increases loading and maintenance doses to similar range as ex-U.S. expansion cohortsNOVATO, Calif., May 17, 2023 Ultragenyx Pharmaceutical Inc. today announced that the. | May 17, 2023

Jeff blakeJoshua higaScott stromattContacts ultragenyx pharmaceutical incExchange commissionUltragenyx pharmaceutical incDrug administrationOrphan drug designationRare pediatric disease designationFast track designationLooking statementsPrivate securities litigation reform actQuarterly reportInvestor relationsUltragenyx pharmaceutical inc stock exchangePress release

Ultragenyx Pharma (RARE) Receives FDA Agreement to Expand Ongoing Global Phase 1/2 Trial Evaluating GTX-102

Ultragenyx Pharma (RARE) Receives FDA Agreement to Expand Ongoing Global Phase 1/2 Trial Evaluating GTX-102
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Joshua higaScott stromattJeff blakeContacts ultragenyx pharmaceutical incDrug administrationUltragenyx pharmaceutical incExchange commissionOrphan drug designationRare pediatric disease designationFast track designationLooking statementsPrivate securities litigation reform actQuarterly reportInvestor relations

Ultragenyx Stock Is A Rare Disease Alpha Player (NASDAQ:RARE)

Ultragenyx is a leader in rare diseases, with 4 approved products and 6 pipeline programs. They have $1bn in cash. See why RARE stock is a long-term hold.

Scott stromattMont blancMereo biopharmaGenetx ultragenyxLinked hypophosphatemiaInduced osteomalaciaFamilial hypercholesterolemiaChain fatty acid oxidation disordersDebrancher deficiencyChief medical officerOvid therapeuticsSmall molecule therapiesNeurodevelopmental disorder angelmanEmerging geneNeurodevelopmental disorder angelman syndromeOsteogenesis imperfecta

GeneTx and Ultragenyx Announce Presentations at Upcoming 2021 FAST Global Summit & Gala

GeneTx and Ultragenyx Announce Presentations at Upcoming 2021 FAST Global Summit & Gala
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United statesRush universityJoshua higaElizabeth berry kravisScott stromattScott dindotCarolyn wangEmil kakkisGenetx biotherapeuticsTexasam universityUltragenyx pharmaceutical incDevelopment of rare disease therapiesDrug administrationNeurological sciencesExchange commissionTranslational research

GeneTx and Ultragenyx Receive Approval from U K Regulatory Agency to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome

GeneTx and Ultragenyx Receive Approval from U K Regulatory Agency to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome
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Scott stromattCarolyn wangGenetx biotherapeuticsUltragenyx pharmaceutical incExchange commission onDrug administrationExchange commissionContactsultragenyx pharmaceutical inc investors joshua higaHealth canadaHealthcare products regulatory agencyUltragenyx pharmaceuticalClinical trial applicationChief medical officerOrphan drug designationRare pediatric disease designationFast track designation