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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Researchers were Wrong about the Cause of Muscular Dystrophy
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Researchers were Wrong about the Cause of Muscular Dystrophy

For a long time, neuromuscular diseases such as muscular dystrophy, ALS, and inclusion body myopathy were thought to be caused as a result of toxic clumps of proteins such as TDP-43.…

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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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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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Ribitol Could Benefit Patients With Rare Limb Girdle Muscular Dystrophy and Related Diseases

A recent study published in the journal Nature Communications highlights a potential therapy for the treatment of muscular dytrophies and other diseases linked to mutations of the FKRP gene. In the study, the…

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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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This SLP’s Mission to Expand Resources for Those with ALS, Muscular Dystrophy, and More

  According to ASHA.org, when Tiffany Turner graduated, she set out to expand opportunities for fellow speech language pathologists like herself. Turner saw a great need in her community for…

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Is Fetal Gene Therapy the Cure to These Rare Diseases?
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Is Fetal Gene Therapy the Cure to These Rare Diseases?

Scientists are researching the possibility of fetal gene therapies as possible cures for rare diseases, such as Duchenne muscular dystrophy, hemophilia, sickle cell disease, and Gaucher's disease.  With fetal gene…

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For Children With Debilitating Illness, the Wait for Medical Equipment Can be Agonizing
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For Children With Debilitating Illness, the Wait for Medical Equipment Can be Agonizing

According to a story from the Los Angeles Daily News, for children with debilitating rare illnesses like cerebral palsy, epilepsy, or muscular dystrophy, it can often take a long time…

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A Phase 3 Study of an Experimental Gene Therapy for Oculopharyngeal Muscular Dystrophy Will Take Place, Axovant Says

An experimental gene therapy program (called AXO-AAV-OPMD) being developed for the treatment of oculopharyngeal muscular dystrophy has been licensed with exclusive global rights to Axovant Sciences from Benitec Biopharma. There…

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MDA Grants $1,000,000 for Charcot-Marie-Tooth Disease Research
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MDA Grants $1,000,000 for Charcot-Marie-Tooth Disease Research

According to a July 9 report by the Muscular Dystrophy Association (MDA), the MDA awarded a large grant to researchers studying Charcot-Marie-Tooth disease. The grant, totaling over $1,000,000, was awarded…

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Investigational Drug for Facioscapulohumeral Muscular Dystrophy Gets Orphan Drug Designation
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Investigational Drug for Facioscapulohumeral Muscular Dystrophy Gets Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company Acceleron Pharma, which develops TGF-beta based treatments for rare and serious illnesses, recently announced that the U.S. Food and Drug Administration…

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First Hemophilia B Patient Enrolled in Potential Breakthrough Phase III Trial

According to BioPortfolio, the company UniQure has recently announced that the first hemophilia B patient has been enrolled in their Phase III study of the treatment AMT-061, which is an…

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Zebrafish Lab Research Could Produce Solutions for Neurodegenerative Disease Like CMT and MS
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Zebrafish Lab Research Could Produce Solutions for Neurodegenerative Disease Like CMT and MS

According to UVA Today, a University of Virginia professor, Sarah Kucenas, is studying glia cells in the brain of fish that could potentially help spear the way for the creation of…

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