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PTC Therapeutics Pioneers in Rare Disease Treatments

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/

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 a …

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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 valuable …

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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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Upstaza™ Gene Therapy Granted Marketing Authorization for …

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 …

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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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PTC Therapeutics Announces FDA Approval of Evrysdi …

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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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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New Analyses of Translarna™ (ataluren) Data from ACT DMD …

Webprojected regulatory timeline for the NDA; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in the European Economic Area, …

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Huntington's disease

WebHuntington’s disease is a rare, hereditary, genetic disorder of the central nervous system. 1 It is caused by a defective gene. This gene produces a protein, called huntingtin, which is …

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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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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 …

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PTC Therapeutics Shares Positive Interim Data from PIVOT-HD …

Web- Dose-dependent lowering of blood Huntingtin (HTT) protein levels at 12 weeks - - Favorable tolerability profile with no treatment-related serious adverse events …

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Aromatic l-amino acid decarboxylase deficiency

WebAromatic l-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder that affects the brain. 1 It interferes with the way the cells in the nervous system talk to each …

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Navigating Behavioral Health in Duchenne Muscular Dystrophy

WebBy Jonathan Blaize, PhD, Medical Science Liaison, Medical Affairs, PTC. To promote awareness of common behavioral concerns, provide a platform for scientific …

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Results Show Long-Lasting and Holistic Improvements in Children …

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

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PTC Therapeutics Provides Update on EMA Regulatory Review …

Web- CHMP Opinion Now Expected May 2022-. SOUTH PLAINFIELD, N.J., April 14, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today …

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

WebGlenn D. Steele Jr., MD, PHD serves as the Chairman of xG Health Solutions, a health care redesign and optimization company affiliated with Geisinger Health System, from …

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David P. Southwell

WebFormer CEO, TScan Therapeutics. Mr. Southwell has served as a member of our Board since 2005. From October 2018 to March 2023, he served as the Chief Executive Officer, …

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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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