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 […]
Here is a summary of today’s call with PTC’s Patient Advocacy and Commercial Teams Thank you PTC for hosting a call for advocacy leaders this morning to introduce the EMFLAZA team and give us a preview of the upcoming launch of this drug. PTC is committed to bringing treatments to rare diseases where there are […]
After her 10-month-old nephew was diagnosed with Duchenne muscular dystrophy, Karin Gorman was inspired to learn more about the disease and find a way to make a difference in the lives of families and individuals affected by Duchenne. A fatal genetic disease with no cure, Duchenne muscular dystrophy causes muscle deterioration and affects more than […]
If you told someone your child had cancer, they would immediately have a sense of the disease and potentially even a sense of the treatment options. People know about chemo, radiation and surgery. They don’t know much about rare diseases like Duchenne muscular dystrophy, and they are less likely to know that there is no […]
  Haydee Bustos has a passion for helping kids, and through her experience as a physical therapist, she discovered an even deeper desire to help kids with Duchenne muscular dystrophy. A fatal genetic disease, Duchenne muscular dystrophy causes muscle deterioration leaving patients, mostly boys, wheelchair bound by their mid-teens and claims their lives by their […]
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