Australian Government Initiates New Trial Program to Support Individuals Recently Diagnosed with a Rare Disease

Being diagnosed with any serious condition can be scary, overwhelming, and full of unknowns. For those who are diagnosed with a rare disease such as Duchenne muscular dystrophy, spinal muscular atrophy,…

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Company Provides Updates for Phase 3 Duchenne Muscular Dystrophy Trial
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Company Provides Updates for Phase 3 Duchenne Muscular Dystrophy Trial

According to a story from drugs.com, the biopharmaceutical company Catabasis pharmaceuticals recently provided updates on the status of its Phase 3 clinical trial testing its investigational therapy CAT-1004, also known…

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ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial
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ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial

According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy…

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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients
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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients

According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…

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Man With Duchenne Muscular Dystrophy Celebrates 50th Birthday After Grim Prognosis as a Child
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Man With Duchenne Muscular Dystrophy Celebrates 50th Birthday After Grim Prognosis as a Child

  Andrew Taylor celebrated his 50th birthday this year. This is a big year for anyone, but especially for Taylor, who was told he would not make it to the…

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The Patriots Raise Awareness for Sickle Cell Anemia and Duchenne Muscular Dystrophy
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The Patriots Raise Awareness for Sickle Cell Anemia and Duchenne Muscular Dystrophy

This past Sunday, December 2nd, during the Patriot's game against the Minnesota Vikings, 20 players wore customized cleats to spread awareness for various philanthropic causes. This is the third year…

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This Just In: India Never Implemented their Rare Disease Policy as Promised
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This Just In: India Never Implemented their Rare Disease Policy as Promised

We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…

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A Clinical Trial for Gene Therapy is Underway for Muscular Dystrophy!
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A Clinical Trial for Gene Therapy is Underway for Muscular Dystrophy!

Limb-Girdle Muscular Dystrophy or LGMD, is a form of Muscular Dystrophy caused by a mutation which disrupts production of the protein beta-sarcoglycan. There are currently no treatment options for LGMD and…

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Participants in a Duchenne Muscular Dystrophy Clinical Trial Found out the Drug Wasn’t Working on Social Media
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Participants in a Duchenne Muscular Dystrophy Clinical Trial Found out the Drug Wasn’t Working on Social Media

According to a story from nature.com, Amber Sapp's twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne…

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Patient Registry Demonstrates The Effectiveness of Translarna in Duchenne Muscular Dystrophy
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Patient Registry Demonstrates The Effectiveness of Translarna in Duchenne Muscular Dystrophy

According to a story from prnewswire.co.uk, the biopharmaceutical company PTC Therapeutics recently announced the release of initial data from the first drug registry for its product Translarna (ataluren). This registry…

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Pfizer Drops Duchenne Muscular Dystrophy Treatment
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Pfizer Drops Duchenne Muscular Dystrophy Treatment

According to a report by biopharmadive.com, Pfizer announced they will be dropping support for an experimental Duchenne muscular dystrophy treatment. The company intends to see if the treatment may be…

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A Greater Number of Drugs Are Getting Cleared With the FDA’s Accelerated Approval. What Does This Mean?
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A Greater Number of Drugs Are Getting Cleared With the FDA’s Accelerated Approval. What Does This Mean?

According to a story from Boston Health News, The US Food and Drug Administration's Accelerated Approval program is being used to approve a steadily growing number of drugs in recent…

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Clinical Trial for Duchenne Muscular Dystrophy Gene Therapy Restarts After Hold Lifted

According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after the FDA lifted the hold.…

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CureDuchenne has Invested $500,000 In a Company to Advance Gene Therapies

The non-profit organisation CureDuchenne has announced that they have made a $500,000 investment in the company 4D Molecular Therapeutics in order to support the development of gene therapy research. For…

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Cryo-Electron Imaging Provides New Info About Virus Vectors in Gene Therapy
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Cryo-Electron Imaging Provides New Info About Virus Vectors in Gene Therapy

According to a story from EurekAlert!, a collaborative team of researchers from the University of Florida and The Salk Institute are using cryo-electron microscopy in order to more closely examine…

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Two Clinical Studies of an Experimental Drug for Duchenne Muscular Dystrophy Have Been Stopped
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Two Clinical Studies of an Experimental Drug for Duchenne Muscular Dystrophy Have Been Stopped

According to a recent press release, Pfizer Inc. has terminated two on-going clinical trials that were investigating domagrozumab (PF-06252616) as a potential treatment for Duchenne muscular dystrophy. This decision follows…

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The First Patient Has Been Dosed on a Study of a Drug For Duchenne Muscular Dystrophy
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The First Patient Has Been Dosed on a Study of a Drug For Duchenne Muscular Dystrophy

The Australian biopharmaceutical company Antisense Therapeutics has announced that the first patient has been dosed on a Phase 2 clinical trial of the investigational drug ATL1102, which is being developed…

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Experimental Duchenne Muscular Dystrophy Drug Gets Multiple Designations From the FDA
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Experimental Duchenne Muscular Dystrophy Drug Gets Multiple Designations From the FDA

According to a story from Globe Newswire, the biotechnology company Wave Life Sciences recently announced that its investigational product WVE-210201 has received both Rare Pediatric disease designation and Orphan Drug…

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An Experimental Drug For Treating Duchenne Muscular Dystrophy Will be Studied in a Phase 3 Trial, Catabasis Says
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An Experimental Drug For Treating Duchenne Muscular Dystrophy Will be Studied in a Phase 3 Trial, Catabasis Says

Catabasis Pharmaceuticals Inc. has announced plans for a Phase 3 clinical trial called Polaris DMD. It will investigate the experimental drug edasalonexent for the treatment of patients with Duchenne muscular…

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