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Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

09:21 EST 22 Feb 2019 | Speciality Pharma Journal

NOVATO, Calif., Feb. 21, 2019 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced positive longer-term top-line safety and efficacy data from the first, lowest dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated …

Original Article: Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

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Biopharmaceuticals are medical drugs produced using biotechnology. They include proteins (including antibodies), nucleic acids (DNA, RNA or antisense oligonucleotides) and living microorganisms like virus and bacteria where the virulance of viruses and b...