New Investigative Therapy Holds Promise for Retinitis Pigmentosa in Mouse Model

Researchers from Nanoscope, thanks to funding by the NIH, have developed a protein that can effectively restore vision in mice. This study was published in Nature Gene Therapy. The Study…

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X-Linked Retinitis Pigmentosa Investigative Gene Therapy is Looking Promising
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X-Linked Retinitis Pigmentosa Investigative Gene Therapy is Looking Promising

Janssen Pharmaceuticals has an ongoing Phase 1/2 clinical trial for the inherited retinal disease X-linked retinitis pigmentosa (XLRP). They are investigating an adeno-associated gene therapy called RPGR as a therapeutic…

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Gene Therapy Could Cure Irreversible Blindness for X-Linked Retinitis Pigmentosa
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Gene Therapy Could Cure Irreversible Blindness for X-Linked Retinitis Pigmentosa

As reported in Biospace, a new gene therapy has made notable progress towards curing x-linked retinitis pigmentosa, a disease responsible for sight loss in men. Until now, the disease irreversibly…

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The World’s First Editing of DNA in Humans Using CRISPR to Treat Leber Congenital Amaurosis

  Allergan plc, a leading global pharmaceutical company, and its partner, Editas Medicine, Inc. have been given the green light for a clinical trial designed to treat patients with severe…

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Company Cleared to Begin Trials for Experimental Retinitis Pigmentosa Drug

According to a story from globenewswire.com, the drug developer ProQR Therapeutics N.V. has recently announced that the US Food and Drug Administration (FDA) has officially cleared the company's Investigational New…

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Learn About These Rare Eye Diseases to Honor Healthy Vision Month!

May is Healthy Vision Month! Rare disease or not, maintaining healthy eye health should be a priority for all of us. The National Institute of Health has resources for not…

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Enrollment Completed in Achromatopsia and Retinitis Pigmentosa Clinical Trials

ACHM and XLRP Achromatopsia (ACHM) is a rare, inherited retinal disease which causes impaired cone photoreceptor function. Patients experience light sensitivity so extreme that it can cause blindness during the…

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Single Use Gene Therapy Approved in the EU for Leber’s Congenital Amaurosis
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Single Use Gene Therapy Approved in the EU for Leber’s Congenital Amaurosis

According to a story from pm360online.com, the drug developer Novartis has recently announced that the company's gene therapy Luxturna has gained approval in the EU as a treatment for Leber's…

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First Patient Receives Treatment in Retinitis Pigmentosa Clinical Trial

According to a story from BioPortfolio, the biopharmaceutical company GenSight Biologics recently announced that the first patient has received treatment in a Phase I/II clinical trial testing the company's experimental…

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Preliminary Data from a Phase 1/2 Study of a Gene Therapy for X-Linked Retinitis Pigmentosa has been Shared
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Preliminary Data from a Phase 1/2 Study of a Gene Therapy for X-Linked Retinitis Pigmentosa has been Shared

Nightstar Therapeutics has released preliminary data from a Phase 1/2 study of an experimental gene therapy designed to treat X-linked retinitis pigmentosa. For more detailed information you can view the…

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