Rare diseases day/SMD trials/Russo
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Thread: Rare diseases day/SMD trials/Russo

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    Rare diseases day/SMD trials/Russo

    Here's the idea. Today is Rare diseases day and ACTC is conducing trials for SMD (which I understand is rare disease) so it would be appropriate to send out some kind of article to media basically pointing out that ACTC is conducing trials for SMD, released promising preliminary results, finished Cohort I, injected first patient in UK etc..
    Some kind of summary of what is ACTC doing on the rare diseases field.
    I know it's but that's the exact thing which I would do if I'm the one who is doing PR for ACTC...
    Rayden, saxxie, ACTRanger and 1 others like this.

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    Great idea.

    Something similiar to the NIH would be nice of course using Stargardt's and ACT. Its good to see NIH is highlighting eye disease.

    NIH spotlights research for rare eye diseases during Rare Disease Day, February 28, 2012 News Release - National Institutes of Health (NIH)
    National Eye Institute (NEI)
    Tuesday, February 28, 2012 Contact:
    National Eye Institute

    NIH spotlights research for rare eye diseases during Rare Disease Day

    Statement from Paul A. Sieving, M.D., Ph.D., director of the National Eye Institute
    On the fifth annual Rare Disease Day, the National Eye Institute (NEI), a part of the National Institutes of Health, joins patients and organizations around the world to raise awareness of rare diseases, celebrate advances in treatment, and pledge continued support of rare disease research.

    In the United States, a disease is considered rare if it affects fewer than 200,000 Americans. Scientists have identified more than 7,000 rare diseases. Some rare diseases affect only a few hundred people. But considered together, rare diseases affect 25 million Americans, which means about 1 in every 10 people has a rare disease.

    Inherited gene defects account for 80 percent of rare diseases, many of which affect vision. Recent advances in gene technology are illuminating our understanding of the causes of rare diseases and quickening the translation of discoveries into new treatments.

    NEI scientists have successfully used gene therapy to improve vision in people with Leber congenital amaurosis, a rare genetic disorder that causes blindness through the degeneration of photoreceptor cells — the rods and cones in the layer of tissue in the back of the eye called the retina. Gene therapy helps restore gene function by inserting copies of normal, functioning genes into cells. Although vision gains were modest, successful demonstration of the strategy holds promise for treating other degenerative retinal disorders such as retinitis pigmentosa (RP). In January 2012, NEI scientists successfully used gene therapy to treat dogs with a condition similar to RP and are making plans to test the therapy in humans.

    People with rare diseases, and their families, have unique challenges beginning with obtaining an accurate diagnosis, which is often delayed due to limited knowledge and awareness in the medical community.

    EyeGENE, an NEI-sponsored collaborative network for ophthalmic research, is helping diagnose patients with eye-related genetic diseases while enabling researchers to study and characterize these rare conditions. Through a network of federally certified molecular diagnostic testing labs, patients who participate in eyeGENE gain access to free genetic testing, which is often expensive and not covered by health insurance. EyeGENE currently offers testing, at labs across the country, for more than two dozen rare genetic conditions. In exchange for free genetic testing, participants contribute their clinical and genetic information to the confidential eyeGENE registry. As researchers develop potential treatments, eyeGENE will help facilitate patient recruitment for clinical trials.

    To help people living with low vision and blindness maintain or increase their independence, the NEI funds the development of assistive technologies and devices through Small Business Innovation Research awards and other grants. For example, Second Sight, a major recipient of NEI funding, has successfully developed a retinal prosthesis — essentially an externally worn digital camera wirelessly coupled to an electrode grid implanted on the retina inside the eye — that restores limited vision to people with RP.

    Rare but Strong Together is the 2012 Rare Disease Day slogan. The NEI stands strong with rare disease patients and their families and is committed to providing research support and leadership for finding new treatments and cures for rare eye-related diseases.

    During Rare Disease Day, NIH is holding a daylong symposium about rare disease research. For more information about the symposium, visit NIH Office of Rare Diseases Research (ORDR) - Rare Disease Day.

    For more information about Rare Disease Day, visit the NIH Office of Rare Disease Research at NIH Office of Rare Diseases Research (ORDR).

    The National Eye Institute, part of the National Institutes of Health, leads the federal government’s research on the visual system and eye diseases. NEI supports basic and clinical science programs that result in the development of sight-saving treatments. For more information, visit National Eye Institute [NEI], of the U.S. National Institutes of Health.

    About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit National Institutes of Health (NIH).

    NIH...Turning Discovery Into Health

    Home > News & EventsSubscribe to receive future NIH news releases.
    Last edited by Invest; 02-29-2012 at 06:34 AM.


    "Dry AMD is a form of macular degeneration with no approved drug treatment available to date,” said Dr. Carl Regillo who led the surgery. ACT's hESC-derived RPE cells could address the unmet medical need of combating dry AMD and other macular degenerations such as Stargardt's disease. We are looking forward to analysing the safety and efficacy data."