NPR said perhaps one million for both eyes. -----Original Message----- From: viphone@googlegroups.com [mailto:viphone@googlegroups.com] On Behalf Of Mohib Anwar Rafay Sent: Friday, December 22, 2017 1:13 AM To: viphone@googlegroups.com Subject: Re: Attention to people with RP
just wondering what will be the expensis incurred for this treatment? On 12/21/17, Marie <magpie...@gmail.com> wrote: > I am including some information I received yesterday concerning a gene > therapy treatment which has been approved by the FDA for people with > Retinitis Pigmentosa. I am sending it to all the lists I currently belong to > because it needs to get to as many RP sufferers as possible. > Marie > Foundation Fighting Blindness Celebrates Historic FDA Approval of > > First Gene Therapy to Treat Blindness > > Foundation’s early investment in LUXTURNA™ boosts vision-restoring treatment > for people with RPE65 mutations and will help advance other gene therapies > currently in development. > > (Columbia, MD) — Today’s U.S. Food and Drug Administration (FDA) approval of > voretigene neparvovec, to be marketed as LUXTURNA, will be life-changing for > patients with vision loss due to mutations in the RPE65 gene and a watershed > moment for the inherited retinal disease field, says the Foundation Fighting > Blindness. The Foundation was an important early investor in LUXTURNA, > providing $10 million in critical seed funding for the therapy. > > The groundbreaking treatment is the first gene therapy for the eye and for > any inherited disease to be approved by the FDA. The treatment restores > vision by delivering working copies of the RPE65 gene directly into the > retina, thereby compensating for the nonfunctional, mutated genes. > > “We are thrilled for the patients whose lives will change dramatically > because of this treatment,” says David Brint, Foundation Fighting Blindness > chairman. “We are also pleased to have this concrete example of the strength > of the Foundation’s strategy of identifying and investing early in promising > treatments. Doing so helps attract industry investment that can usher > promising treatments through clinical trials and ultimately FDA approval.” > > LUXTURNA is the result of more than two decades of research and development > at the University of Florida, the University of Pennsylvania, Children’s > Hospital of Philadelphia, and Spark Therapeutics. The Foundation Fighting > Blindness’ seed investment allowed researchers to take the therapy through > the early investigational stages critical to any treatment development. > > “LUXTURNA will be life-changing for people with an inherited retinal disease > caused by RPE65 mutations. For them, the treatment means alife of > independence. Also important is the momentum this approval provides to other > gene-based therapies — for the eye and other diseases — now in the clinic,” > says Benjamin Yerxa, PhD, Foundation CEO. > > An additional noteworthy milestone is the demonstrated value of a new > clinical endpoint devised by the Spark Therapeutics team to measure > LUXTURNA’s impact. The new measure, a multi-luminance mobility test > (informally called the maze), measured the impact of the treatment beyond > the traditional visual acuity measure — the eye chart. This new clinical > endpoint moves vision measures beyond the eye chart, which is particularly > significant for people with low or no vision. > > Spark Therapeutics, which holds the biologics license for LUXTURNA and > conducted the clinical trials that showed its safety and efficacy, will also > manage the treatment rollout. Spark has announced that in order to ensure > the treatment is safely administered, it will only be available through a > small number of centers of clinical excellence across the country. Spark has > also expressed its commitment to educating third-party payers about the > value of LUXTURNA and to working to help ensure treatment access to all > eligible patients. > > Anyone in need of more information about LUXTURNA should contact Spark > Therapeutics at 1-833-SPARK-PS (833-772-7577). Another resource for > information is Spark’s website: www.Sparktx.com. > > > Foundation Fighting Blindness Celebrates Historic FDA Approval of > > First Gene Therapy to Treat Blindness > > Foundation’s early investment in LUXTURNA™ boosts vision-restoring treatment > for people with RPE65 mutations and will help advance other gene therapies > currently in development. > > (Columbia, MD) — Today’s U.S. Food and Drug Administration (FDA) approval of > voretigene neparvovec, to be marketed as LUXTURNA, will be life-changing for > patients with vision loss due to mutations in the RPE65 gene and a watershed > moment for the inherited retinal disease field, says the Foundation Fighting > Blindness. The Foundation was an important early investor in LUXTURNA, > providing $10 million in critical seed funding for the therapy. > > The groundbreaking treatment is the first gene therapy for the eye and for > any inherited disease to be approved by the FDA. The treatment restores > vision by delivering working copies of the RPE65 gene directly into the > retina, thereby compensating for the nonfunctional, mutated genes. > > “We are thrilled for the patients whose lives will change dramatically > because of this treatment,” says David Brint, Foundation Fighting Blindness > chairman. “We are also pleased to have this concrete example of the strength > of the Foundation’s strategy of identifying and investing early in promising > treatments. Doing so helps attract industry investment that can usher > promising treatments through clinical trials and ultimately FDA approval.” > > LUXTURNA is the result of more than two decades of research and development > at the University of Florida, the University of Pennsylvania, Children’s > Hospital of Philadelphia, and Spark Therapeutics. The Foundation Fighting > Blindness’ seed investment allowed researchers to take the therapy through > the early investigational stages critical to any treatment development. > > “LUXTURNA will be life-changing for people with an inherited retinal disease > caused by RPE65 mutations. For them, the treatment means alife of > independence. Also important is the momentum this approval provides to other > gene-based therapies — for the eye and other diseases — now in the clinic,” > says Benjamin Yerxa, PhD, Foundation CEO. > > An additional noteworthy milestone is the demonstrated value of a new > clinical endpoint devised by the Spark Therapeutics team to measure > LUXTURNA’s impact. The new measure, a multi-luminance mobility test > (informally called the maze), measured the impact of the treatment beyond > the traditional visual acuity measure — the eye chart. This new clinical > endpoint moves vision measures beyond the eye chart, which is particularly > significant for people with low or no vision. > > Spark Therapeutics, which holds the biologics license for LUXTURNA and > conducted the clinical trials that showed its safety and efficacy, will also > manage the treatment rollout. Spark has announced that in order to ensure > the treatment is safely administered, it will only be available through a > small number of centers of clinical excellence across the country. Spark has > also expressed its commitment to educating third-party payers about the > value of LUXTURNA and to working to help ensure treatment access to all > eligible patients. > > Anyone in need of more information about LUXTURNA should contact Spark > Therapeutics at 1-833-SPARK-PS (833-772-7577). Another resource for > information is Spark’s website: www.Sparktx.com. > > > --- > This email has been checked for viruses by Avast antivirus software. > https://www.avast.com/antivirus > > -- > The following information is important for all members of the V iPhone > list. > > If you have any questions or concerns about the running of this list, or if > you feel that a member's post is inappropriate, please contact the owners or > moderators directly rather than posting on the list itself. > > Your V iPhone list moderator is Mark Taylor. Mark can be reached at: > mk...@ucla.edu. Your list owner is Cara Quinn - you can reach Cara at > caraqu...@caraquinn.com > > The archives for this list can be searched at: > http://www.mail-archive.com/viphone@googlegroups.com/ > --- > You received this message because you are subscribed to the Google Groups > "VIPhone" group. > To unsubscribe from this group and stop receiving emails from it, send an > email to viphone+unsubscr...@googlegroups.com. > To post to this group, send email to viphone@googlegroups.com. > Visit this group at https://groups.google.com/group/viphone. > For more options, visit https://groups.google.com/d/optout. > -- Mohib Anwar Rafay Phone: +919 555 555 765 -- The following information is important for all members of the V iPhone list. If you have any questions or concerns about the running of this list, or if you feel that a member's post is inappropriate, please contact the owners or moderators directly rather than posting on the list itself. Your V iPhone list moderator is Mark Taylor. Mark can be reached at: mk...@ucla.edu. Your list owner is Cara Quinn - you can reach Cara at caraqu...@caraquinn.com The archives for this list can be searched at: http://www.mail-archive.com/viphone@googlegroups.com/ --- You received this message because you are subscribed to the Google Groups "VIPhone" group. To unsubscribe from this group and stop receiving emails from it, send an email to viphone+unsubscr...@googlegroups.com. To post to this group, send email to viphone@googlegroups.com. Visit this group at https://groups.google.com/group/viphone. For more options, visit https://groups.google.com/d/optout. -- The following information is important for all members of the V iPhone list. If you have any questions or concerns about the running of this list, or if you feel that a member's post is inappropriate, please contact the owners or moderators directly rather than posting on the list itself. Your V iPhone list moderator is Mark Taylor. Mark can be reached at: mk...@ucla.edu. 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