Akron Area Family Overwhelmed with Gratitude by Support Shown for Son with Chiari Malformation
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Akron Area Family Overwhelmed with Gratitude by Support Shown for Son with Chiari Malformation

By Caitlin Seida from In The Cloud Copy Jacob Myers was an active 15-year-old until last spring. The honor roll student, who also sang in the school choir and participated…

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Gene Therapy Being Assessed for Treatment of X-Linked Severe Combined Immunodeficiency
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Gene Therapy Being Assessed for Treatment of X-Linked Severe Combined Immunodeficiency

  Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…

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Experimental Myelodysplastic Syndrome Treatment Receives Breakthrough Therapy Designation

  A drug created by Agios Pharmaceuticals, TIBSOVO, was recently granted the Breakthrough Therapy designation by the FDA for the treatment of relapsed or refractory myelodysplastic syndrome (MDS) with an…

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FDA Grants Orphan Drug Designation for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency
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FDA Grants Orphan Drug Designation for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency

Dicerna Pharmaceuticals works in ribonucleic interference (RNAi) therapies as treatments of rare diseases. They have recently been granted the orphan drug designation by the European Commission for their drug, DCR-A1AT.…

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Treatment for Duchenne Muscular Dystrophy with Rare Mutations Accepted Under Accelerated Approval
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Treatment for Duchenne Muscular Dystrophy with Rare Mutations Accepted Under Accelerated Approval

According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved the drug golodirsen (marketed as Vyondys 53) as a treatment for Duchenne muscular dystrophy…

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Trial Application for Experimental Cannabinoid Epidermolysis Bullosa Treatment Gains Approval
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Trial Application for Experimental Cannabinoid Epidermolysis Bullosa Treatment Gains Approval

According to a story from prnewswire.com, the biopharmaceutical company InMed Pharmaceuticals, Inc. has recently announced that that company's Clinical Trial Application has been approved in the Netherlands. This approval will…

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With Clinical Hold Lifted, Abeona Moves Forward with Recessive Dystrophic Epidermolysis Bullosa Trial
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With Clinical Hold Lifted, Abeona Moves Forward with Recessive Dystrophic Epidermolysis Bullosa Trial

According to a story from Benzinga, the drug company Abeona Therapeutics, Inc. has recently announced plans to move forward with its phase 3 clinical trial following the lifting of the…

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