steroid

Cranbury Pharmaceuticals (Tris Pharma) Receives FDA Approval for First Generic Version of Emflaza® (deflazacort) for Duchenne

Cranbury Pharmaceuticals, a subsidiary of Tris Pharma, today announced the U.S. Food and Drug Administration (FDA) approved the Abbreviated New Drug Application (ANDA) for the first generic version of Emflaza® oral suspension (deflazacort) for the…

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Take Part in the Duchenne Steroid Patient Experience Survey

Patient experience data is a critical tool for advancing drug development in Duchenne and for regulatory decision making. Steroids have been part of care standards for over two decades, however, there is a lack of…

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August 16, 2019

ICER Releases Report; PPMD Issues Open Letter to Payers

Last night, the Institute for Clinical and Economic Review (ICER) released their final report assessing three Duchenne products: Emflaza, EXONDYS 51, and golodirsen. This report integrates all public comments, stakeholder engagement, and the Roundtable proceedings…

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PTC Therapeutics Update on Specialty Pharmacy Transition

PTC Therapeutics has shared an update regarding the transitioning of patients that are taking EMFLAZA® (deflazacort) to a new specialty pharmacy, Accredo. Read the Update from PTC: PTC Therapeutics Update on Specialty Pharmacy Transition In…

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PTC Receives FDA Approval for Expansion of EMFLAZA® (deflazacort) Labeling to Include Patients 2-5 Years of Age

PTC Therapeutics announced that they have received FDA approval for the expansion of the EMFLAZA® (deflazacort) labeling to include patients 2-5 years of age. Emflaza was first approved by the FDA in February 2017 for…

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October 31, 2018

WATCH: An Update on Corticosteroids – Webinar Recording

PPMD and Dr. John Brandsema hosted a webinar on October 24, 2018 to discuss the current guidelines for steroid use in Duchenne muscular dystrophy, emerging data, and best practices. If you missed the live event,…

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