08 May
  • By Alicia Clough
  • Cause in

EMFLAZA Launch Plan

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 […]

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08 May
  • By Alicia Clough
  • Cause in

Duchenne Community Letter from PTC Therapeutics

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 […]

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25 Apr
  • By Alicia Clough
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HOPE Clinical Trial Provides Hope for Duchenne Families

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 […]

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20 Apr
  • By Alicia Clough
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Duchenne Community Message From PTC Therapeutics on Close of Emflaza Acquisition

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 […]

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04 Apr
  • By Alicia Clough
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Anthem Please Provide Access to Approved Duchenne Treatment

(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 […]

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24 Mar
  • By Alicia Clough
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Letter to Duchenne Community from PTC Therapeutics

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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21 Mar
  • By Alicia Clough
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PTC-Duchenne Advocacy Community Call, March 21, 2017

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 […]

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22 Feb
  • By cure.duchenne
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Physical Therapist Haydee Bustos is a Pillar of Strength in the Duchenne Community

  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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13 Feb
  • By cure.duchenne
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An Open Letter to the Duchenne Community

  CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community Dear Members of the Duchenne Community: I want to thank you all for your support and encouragement over the last several years as Marathon worked to bring EMFLAZA™ (deflazacort) through the […]

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13 Feb
  • By cure.duchenne
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An Open Letter to the Duchenne Community

  CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community  Dear Members of the Duchenne Community: I want to thank you all for your support and encouragement over the last several years as Marathon worked to bring EMFLAZA™ (deflazacort) through the […]

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