Today, PTC Therapeutics hosted a call for the Duchenne community to announce pricing and services for their launch of EMFLAZA, the commercial name for deflazacort.  The announced price will be  $35,000/year for a 25kg patient. This is the net pricing to payors.  PTC is committed to provide access and availability of EMFLAZA at no or […]
Mother’s Day is a reminder to love our moms a little extra and be thankful for all that they do for us. For boys with Duchenne, it’s a reminder of how their mothers impact their lives in the largest ways and with the smallest gestures. The boys say it best. Here’s some of their stories: […]
May 8, 2017   Dear Duchenne Families, Today we shared our plans for the launch of EMFLAZA™ (deflazacort), the first FDA-approved corticosteroid for Duchenne muscular dystrophy patients age 5 and older, regardless of the genetic mutation. We are excited to launch EMFLAZA in the U.S., and we will begin making the product commercially available to […]
CureDuchenne is pleased to see the positive preliminary data from Capricor Therapeutics on their HOPE clinical trial in Duchenne muscular dystrophy. CureDuchenne Ventures helped fund this clinical trial to address cardiac failure, the primary cause of death for Duchenne patients.  This data provides hope to  families with Duchenne. Capricor Therapeutics announced positive top-line results from […]
April 20, 2017   Dear Duchenne Community, Today we announced that we have completed our acquisition of Emflaza™ (deflazacort), the first FDA-approved, anti-inflammatory therapy for Duchenne Muscular Dystrophy (DMD) patients age 5 and older regardless of the genetic mutation. We are pleased the acquisition completed ahead of schedule, following early conclusion of the antitrust review […]
NVC Consulting, a market research firm, is looking for patients and caregivers to participate in a paid market research study regarding the treatment of patients with Duchenne Muscular Dystrophy on behalf of a sponsoring pharmaceutical company.  Please see the information below if you are interested in participating. Duchenne Muscular Dystrophy Patients/Caregivers Wanted Seeking DMD patients […]
Physical Therapist Elina Gonzalez’s intuition has been a driving force in her career. During one of her early cases, a child presented signs of muscle weakness and underdevelopment, but remained undiagnosed. Elina took the time to carefully examine her patient and consult the child’s father with additional questions about the child’s symptoms. Elina’s gut feeling […]
(CureDuchenne letter to Anthem dated March 14) CureDuchenne believes that all those with Duchenne that have a treatment option available to them should have access to the drug.  Our heart breaks every time we hear that an insurance company has denied coverage to a Duchenne patient who could benefit from an approved treatment. CureDuchenne is […]
CureDuchenne is proud to sponsor SB 643 (Pan). SB 643 will add Duchenne to the Genetically Handicapped Persons Program’s (GHPP) list of eligible medical conditions. Duchenne is a rare neuromuscular disorder characterized by progressive symmetric muscle weakness and degeneration stemming from the progressive loss of contractile function. Patients in California with Duchenne currently receive specialized […]
We are happy to share the following letter to the Duchenne Community from Stu Peltz, CEO PTC Therapeutics.  March 24, 2017   Dear Duchenne Community, We were very excited to announce on March 16, 2017 that PTC Therapeutics entered into an agreement to acquire the FDA approved steroid for Duchenne muscular dystrophy, Emflaza™ (deflazacort) from […]
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