28 Jun
  • By Alicia Clough
  • Cause in

Access to Exondys 51 in Texas

If you live in Texas, are amendable to exon skipping 51, and you are having difficulty accessing Exondys 51, you or your provider may file a complaint to HPM_Complaints@hhsc.state.tx.us.  Exondys 51 is covered by STAR Kids, Texas’s Medicaid managed care program.   Type of information needed: Patient identification number Reference identification number Health plan Provider […]

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27 Jun
  • By Alicia Clough
  • Cause in

Collaboration to Fund CRISPR/Cas9 for Duchenne

Duchenne organizations are invited to fund CRISPR/Cas9 research at Exonics Therapeutics in a venture philanthropy collaboration. We are making progress on treatments for Duchenne, but a cure is the goal our hearts long for. With the innovative CRISPR/Cas9 research being conducted by Dr. Eric Olson at the University of Texas Southwestern Medical Center, there is […]

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16 Jun
  • By Alicia Clough
  • Cause in

Determination and Duchenne – Inspiration from my son on Father’s Day

Each year on Father’s Day, I like to take a moment and reflect on the memories I have with my family, specifically with my son Hawken. Being a father to Hawken has been, and always will be, the most meaningful role in my life. He has made me a proud father. His determination, kindness, and […]

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15 Jun
  • By Alicia Clough
  • Cause in

Dear Dad – A Father’s Day Message from Sons to Dads

Father’s Day is the perfect time to reflect on all that our fathers do for us. For boys with Duchenne, it’s a day that they get to cherish their fathers a little more for their exemplary strength and courage as they battle side by side with their sons against this incurable disease. The boys put […]

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13 Jun
  • By Alicia Clough
  • Cause in

Beyond the Lab – CureDuchenne Ventures Helps Accelerate Academic Research into Promising Medical Care for Patients

Academia has long been an incubating source of incredible medical breakthroughs. Sometimes it takes a little push and risk-taking to take that progressive research out of the lab and into a business environment to achieve life-changing treatments for patients. Such is the case for a promising new technology that has the potential to help 80% […]

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09 Jun
  • By Alicia Clough
  • Cause in

Meet Alex Chiabai – First Boy in Canada to Begin Drisapersen Redosing

Five years ago, 12-year-old Alex Chiabai was one of the first boy in Canada to begin taking drisapersen, a promising drug that provided a glimmer of hope for Alex and his family. After stopping and starting the drug several times over the past few years, Alex once again has the distinction of being the first […]

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06 Jun
  • By Alicia Clough
  • Cause in

PTC /Ataluren – FDA AdCom Meeting September 28, 2017

CureDuchenne is pleased to see that the FDA will hold an Advisory Committee Meeting (AdCom) for PTC Therapeutics’s drug Ataluren.  We are hopeful for a full and fair review which will prioritize the patient and physician voice and experience. Rare diseases are complicated and regulators need to hear about the real world benefit that patients […]

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01 Jun
  • By Alicia Clough
  • Cause in

CD Access Began Redosing Drisapersen in Canada

For Doug Penner, now 14, being part of the original drisapersen trial was a hopeful time; the drug enabled him to go on four-mile walks, climb stairs and even jump. For a 9-year old boy with Duchenne this was remarkable. For Doug’s parents this was a time of enormous hope. Created by Prosena, drisapersen was […]

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17 May
  • By Alicia Clough
  • Cause in

Groundbreaking Research on Gene Editing for Duchenne Presented at ASGCT

The 2017 American Society of Gene & Cell Therapy took place in Washington DC last week. The Duchenne community was treated to a wealth of data from projects utilizing gene editing and gene therapy to treat Duchenne muscular dystrophy. On Wednesday, May 10, the well-attended session entitled “Gene Therapies for Musculoskeletal Diseases was chaired by […]

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16 May
  • By Alicia Clough
  • Cause in

EMFLAZA Bridge Program and Prescription Start Form

PTC is committed to getting patients onto EMFLAZA as quickly as possible, without interruption of therapy. For patients that are currently accessing EMFLAZA through Master Pharmacy importation from outside of the US, they may have immediate access to a “Bridge Program” that will provide EMFLAZA free of charge while their insurance status is being investigated. […]

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