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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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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Treatment for Type 2E Limb Girdle Muscular Dystrophy Gets Rare Pediatric Drug Designation
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Treatment for Type 2E Limb Girdle Muscular Dystrophy Gets Rare Pediatric Drug Designation

According to a story from Business Wire, the gene therapy company Myonexus Therapeutics announced that the U.S. FDA has given Rare Pediatric Disease Designation for its gene therapy candidate MYO-101.…

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An Experimental Drug for Duchenne Muscular Dystrophy Has Been Awarded Orphan Drug Status by the F.D.A.
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An Experimental Drug for Duchenne Muscular Dystrophy Has Been Awarded Orphan Drug Status by the F.D.A.

The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug Sarconeos, developed by the company Biophytis, for the treatment of Duchenne muscular dystrophy. The…

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An Experimental Treatment for FSHD Has Been Awarded Fast Track Designation by the FDA
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An Experimental Treatment for FSHD Has Been Awarded Fast Track Designation by the FDA

Acceleron Pharma has just received Fast Track designation from the US Food and Drug Administration (FDA) for their experimental treatment ACE-083 designed to treat patients with facioscapulohumeral muscular dystrophy (FSHD).…

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The UK Parliament Met With a Mother Advocating For Duchenne Muscular Dystrophy Patients
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The UK Parliament Met With a Mother Advocating For Duchenne Muscular Dystrophy Patients

Phillippa Farrant, whose son, Daniel, has been diagnosed with Duchenne muscular dystrophy (DMD), recently appeared in Parliament in the UK to fight for improved access to care and treatment for…

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How a Local Community Supports a Child with Duchenne Muscular Dystrophy
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How a Local Community Supports a Child with Duchenne Muscular Dystrophy

The phrase “it takes a village” gets tossed around a lot. But in Codicote, that’s what’s happening. Fundraising events and challenges are being held left and right. All of them…

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A Potential Gene Therapy for Duchenne Muscular Dystrophy Has Just Begun Patient Testing
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A Potential Gene Therapy for Duchenne Muscular Dystrophy Has Just Begun Patient Testing

Pfizer has just begun a medical trial for a potential gene therapy treatment for Duchenne muscular dystrophy, and the first patient has been dosed. Click here to read the original…

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Company Continues to Back an Experimental Therapy for Duchenne Muscular Dystrophy Despite Clinical Hold
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Company Continues to Back an Experimental Therapy for Duchenne Muscular Dystrophy Despite Clinical Hold

According to a story from genengnews.com, the pharmaceutical company Solid Biosciences is pledging to continue developing its investigation drug candidate SGT-001 for Duchenne muscular dystrophy. This is despite its clinical…

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Active Man Sets an Example of The Success of Mechanical Ventilation Technology
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Active Man Sets an Example of The Success of Mechanical Ventilation Technology

According to a story from Business Wire, there was a time when having to use a mechanical ventilator in order to breathe meant being confined at the hospital in an…

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Investigational Therapy for Duchenne Muscular Dystrophy is Seeking Orphan Drug Designation
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Investigational Therapy for Duchenne Muscular Dystrophy is Seeking Orphan Drug Designation

According to a story from financialbuzz.com, the biotechnology company BIOPHYTIS has submitted documentation for orphan drug designation from the European Medicines Agency (EMA). This designation will be for Sarconeos, a…

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A Mother and Daughter in India With Muscular Dystrophy Have Started A Hunger Strike To Demand Treatment and Support
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A Mother and Daughter in India With Muscular Dystrophy Have Started A Hunger Strike To Demand Treatment and Support

According to a story from aninews.in, mother Shashi Mishra and her daughter Anamika Mishra have staged a hunger strike in a desperate move to receive treatment for their muscular dystrophy.…

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