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
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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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11 May
  • By Alicia Clough
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Dr. Olson Presents on Gene Editing

Today, Dr. Eric Olson, PhD, Chair of Molecular Biology at UT Southwestern, was honored to give a presentation at the American Society of Gene & Cell Therapy (ASGCT) 2017 meeting in Washington DC for the George Stamatoyannopoulos Lecture series. Dr. Olson presented new research showing how gene editing technologies like CRISPR/Cas9 and Cpf-1 can be […]

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08 May
  • By Alicia Clough
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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
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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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