CONTEXT: Retrospective chart review of patients with dystrophinopathies shows benefits of switching medications to delay disease progression.
IMPACT: Medium
READ TIME: 2 mins
Quality Level Mean [1 – 10]: 7
1. “- Desire to slow disease progression and tolerability issues were the primary reasons for switching from prednisone to EMFLAZA® (deflazacort) -. – Delayed disease progression and improved benefit-risk reported during the 6-month average follow up after switching to EMFLAZA -. SOUTH PLAINFIELD, N.J., March 15, 2021 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from a real-world study of patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) who switched from prednisone to EMFLAZA® (deflazacort) following the FDA approval.”
2. “Physicians ranked desire to slow disease progression, tolerability and caregiver or patient request as the most common motivations for switching from prednisone to EMFLAZA.”
3. “To the extent recorded in medical records, Clinical Global Impression (CGI) of Improvement scores were collected at the clinic visit closest to steroid switching during prednisone treatment, and the last available clinic visit during EMFLAZA treatment.”
4. “”Results from the real-world chart review presented today support the potential of EMFLAZA to alter the natural history of Duchenne muscular dystrophy, demonstrating its capability to slow progression of the disease and improve benefit-risk,” said Dr. Susan Apkon, Investigator, Chief Pediatric Rehabilitation, Vice-Chair Department of Physical Medicine and Rehabilitation, Fischahs Chair in Pediatric Rehabilitation, Children’s Hospital of Colorado.”
5. “About EMFLAZA® (deflazacort). EMFLAZA® is indicated for the treatment of Duchenne muscular dystrophy in patients two years of age and older.”
Source URL: https://finance.yahoo.com/news/ptc-presents-results-real-world-120000671.html