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PTC Therapeutics Pioneers in Rare Disease …

WEBPTC is an established global biopharmaceutical company that delivers transformative therapies for people living with rare diseases. For 25 years, we have been harnessing …

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URL: https://www.ptcbio.com/

PTC Therapeutics

WEBI am extremely proud to have the opportunity to lead PTC’s experienced and dedicated team into its next quarter century as we advance our pioneering science to develop …

Category:  Health Go Health

Upstaza™ Gene Therapy Granted Marketing Authorization …

WEBUpstaza is a one-time gene replacement therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis …

Category:  Health Go Health

PTC Therapeutics Announces FDA Approval of …

WEBEvrysdi will be marketed in the United States by Genentech , a member of the Roche Group, and received an orphan drug designation from FDA in 2017.

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Results Show Long-Lasting and Holistic Improvements in …

WEBSOUTH PLAINFIELD, N.J., Sept. 29, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented a new analysis of five-year results that shows its …

Category:  Health Go Health

Duchenne muscular dystrophy

WEBWhat is Duchenne Muscular Dystrophy?. Muscular dystrophies involve progressive muscle wasting and weakness and are caused by a mutation in the DNA that results in either …

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PTC Therapeutics Receives Positive CHMP Opinion for …

WEB"We are thrilled with the positive opinion from the CHMP, and are eager to bring Upstaza to patients living with AADC deficiency," said Stuart W. Peltz, Ph.D., Chief Executive …

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PKU disease overview

WEBThe estimated global prevalence of PKU is 1 in 23,930 live births 5 and in the US there are thought to be ~16,500 people living with PKU. 6 . Prevalence data was estimated for 64 …

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Healthcare Providers Resources

WEBOur medical team is committed to providing accurate, up-to date and balanced scientific information that enables health care providers to make informed decisions. If you have …

Category:  Medical Go Health

PTC Therapeutics and AADC Family Network Announce First-Ever …

WEBSOUTH PLAINFIELD, N.J., Oct. 22, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) and the AADC Family Network today announced that Oct. 23, 2020, …

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Why It’s Imperative to Continue to Improve the Care of Adults …

WEBIndividuals living with Duchenne are now living longer lives thanks to marked improvements in disease management. 1 This is a positive development, but it also presents new …

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Health and Human Services Expert Mary L. Smith Joins PTC …

WEBSOUTH PLAINFIELD, N.J., June 9, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the election of Mary L. Smith to the Company's …

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Matthew B. Klein, M.D., M.S., F.A.C.S.

WEBMatthew B. Klein, M.D., M.S., F.A.C.S. Chief Executive Officer Dr. Klein has been our Chief Executive Officer since March 2023. Dr. Klein joined the Company in October 2019 as …

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Professional Society for Health Economics and Outcomes Research …

WEBThe Professional Society for Health Economics and Outcomes Research, better known as ISPOR (formerly the International Society for Pharmacoeconomics and Outcomes …

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Glenn D. Steele, Jr., M.D., Ph.D.

WEBDr. Steele has served as a member of our Board since 2015. Dr. Steele has served as the Chairman of xG Health Solutions, a health care redesign and optimization company …

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Insightful Moments

WEBVideos will open in a video player, so you can view them directly in your browser. You can navigate between the Education Series’ by clicking the category button. We are here to …

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Preliminary Data from FIREFISH trial in Type 1 SMA Infants …

WEB1 Kolb S, et al. Amer Neuro Assoc 2017; 883-891 2 Finkel R, et al. Amer Acad Neur 2014; 810-817 About Spinal Muscular Atrophy (SMA) Spinal muscular atrophy (SMA) is a …

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PTC Therapeutics Receives Complete Response Letter for …

WEBOctober 25, 2017 PTC Therapeutics Receives Complete Response Letter for Ataluren's NDA SOUTH PLAINFIELD, N.J., Oct. 25, 2017 /PRNewswire/ -- PTC Therapeutics, Inc. …

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Roadmap to Newborn Screening Changes: Q&A With Judit Illes

WEBRoadmap to Newborn Screening Changes: Q&A With Judit Illes. Judit Illes, Director, Government Affairs & Public Policy at PTC recently contributed to a newly published …

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