The Discovery of 261 New Gene Mutations Which Could Cause Blindness May Lead to More Accurate Diagnoses and New Treatments
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The Discovery of 261 New Gene Mutations Which Could Cause Blindness May Lead to More Accurate Diagnoses and New Treatments

Ala Moshiri from the University of California is both a researcher and an eye doctor. He recently discussed his frustration surrounding the lack of available treatments for so many genetic…

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InformedDNA, Foundation Fighting Blindness Partnership Will Screen Patients for Usher Syndrome, RP Clinical Trials
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InformedDNA, Foundation Fighting Blindness Partnership Will Screen Patients for Usher Syndrome, RP Clinical Trials

ProQR Therapeutics is working to develop unique therapeutic solutions for genetic eye diseases. Currently, the company is evaluating its therapeutic candidate ultevursen within the Phase 2/3 Sirius and Celeste clinical trials; you can read…

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INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 2)
Daniel de Boer. Photo courtesy of: ProQR and FTI Consulting

INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 2)

If you would like to read the first part of this interview, please take a look at Part 1 here. In the first part of the interview, I spoke with…

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INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 1)
Daniel de Boer. Photo courtesy of: ProQR and FTI Consulting

INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 1)

Clinical trials, drug development, and medical research have the potential to change the therapeutic landscape for patients with a wide variety of conditions. At ProQR, founder and CEO Daniel de…

Continue Reading INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 1)
Results Available from GS030 Study for RP in NHP
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Results Available from GS030 Study for RP in NHP

Recently, biopharmaceutical company GenSight Biologics ("GenSight") announced the publication of data from a study evaluating GS030-Drug Products (GS030-DP or "GS030") as a potential treatment option for retinitis pigmentosa (RP). Although…

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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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