Our JumpStart Partner, The Shank2 Foundation, invites you to the their 4th annual SHANK2 Day Conference. It will take place virtually on Sunday, November 10th 2024 rom 8:30 am to 1:30 pm EST. Join them to learn more about SHANK2 and the SHANK2 foundation: https://ow.ly/7zpA50TS5l7
Orphan Disease Center | University of Pennsylvania
研究服务
Philadelphia，Pennsylvania 1,001 位关注者
Assuring access to transformative therapies for all rare disease communities.
关于我们
Mission The Orphan Disease Center will develop transformative therapies using platform technologies that can be deployed across multiple rare diseases. We will emphasize disorders with substantial unmet need independent of their incidence and will strive to assure access to patients of all populations. Impact Each type of orphan disease affects such a small subset of the population, so the need for research and funding in this area is largely unmet. Our Center, the first of its kind, works closely with patient groups and foundations, pharma and biotech, and the academic community. We bring a unique set of programs to the table, enabling us to add value at any stage - from building the initial knowledge base to enabling therapeutic development. Through our grants, Programs of Excellence, JumpStart programs, and a number of new initiatives, the ODC seeks to drive therapeutic development for rare diseases. We help identify and fund the most promising therapeutics while also tackling The focus of the Center lies in partnership and leveraging of resources Uniting investigators and clinicians within Penn, Chop, and internationally who are committed to treating and curing orphan disorders/diseases; Creating resources for the rare disease community to enable discovery and preclinical development of potential therapies, as well as the clinical translation of those efforts; Extending successful approaches developed in one disorder to multiple others, and developing new technologically advanced research services to support this research; Providing a facile means for both small biotech and large pharmaceutical companies to partner with academic researchers in orphan disease research and therapeutic development; Linking academicians to both public and private foundations that support biomedical research for orphan diseases.
- 网站
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https://www.orphandiseasecenter.med.upenn.edu/
Orphan Disease Center | University of Pennsylvania的外部链接
- 所属行业
- 研究服务
- 规模
- 11-50 人
- 总部
- Philadelphia，Pennsylvania
- 类型
- 非营利机构
地点
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主要
获取路线
125 S 31st Street
US，Pennsylvania，Philadelphia，19104-3413
Orphan Disease Center | University of Pennsylvania员工
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Dan Lavery
Chief Scientific Officer, Loulou Foundation & Director, CDKL5 Program of Excellence, Orphan Disease Center
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Samantha Charleston
Director, Programs and Community Engagement at The Orphan Disease Center, University of Pennsylvania
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Cara M. Weismann, Ph.D.
Director, Program of Excellence in MPS & Lysosomal Storage Disease Advanced Therapies, Orphan Disease Center, Gene Therapy Program, University of…
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Caleb Rhodes
MD Candidate at the Perelman School of Medicine
动态
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Orphan Disease Center | University of Pennsylvania转发了
Lysosomal Storage Disease Scientific Colleagues and Patient Advocates, The Orphan Disease Center & the Critical Path for Lysosomal Diseases Consortium are holding a data sharing workshop webinar series! Please provide feedback on data sharing experiences by October 5,2024 https://lnkd.in/eJVXZuWT
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Lysosomal Storage Disease Scientific Colleagues and Patient Advocates, The Orphan Disease Center & the Critical Path for Lysosomal Diseases Consortium are holding a data sharing workshop webinar series! Please provide feedback on data sharing experiences by October 5,2024 https://lnkd.in/eJVXZuWT
Fill | Lysosomal Storage Disease Data Sharing Workshop, Pre-workshop survey
https://forms.office.com/Pages/forms.office.com
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Are you thinking about gene therapy? We want to hear from you! The Working Group on Pediatric Gene Therapy & Medical Ethics (PGTME) wishes to learn from patients, advocates, members of industry, and clinicians about what issues are of paramount importance with regard to pediatric gene therapy clinical trials, past, present, and future. As such, we are convening two groups for open-ended discussions of these topics: Industry and Clinicians: Monday, October 7th, 12-1:15pm ET Patients and Advocates: Wednesday, October 9th, 12-1:15pm ET Each session will be limited to 40 participants. Please note that you may only attend ONE. Please RSVP in advance to the appropriate session to secure your spot! Please share this invitation widely. We look forward to seeing you virtually in October!
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Orphan Disease Center | University of Pennsylvania转发了
President & Founder: Sisters’ Hope Foundation, Rare Disease/Young Onset Dementia Advocate, FDA Appointed Speaker on Caregiving and Gene Therapy, ALSP Expert, Jura Health Advisory Board Member, Advisor FDA-TAP program.
Sisters' Hope Foundation is holding its annual #RunNRoll 5K for a Cure. This year's race will take place virtually and in-person in Lebanon, PA at the #LebanonValleyCraftBrewery with food, drinks, live entertainment by #AddiGrace and #MrGreenGenes very own #JohnnyPhatt and of course, the 5K. We are looking for sponsors for our event and race participants. Sponsorship link:?https://lnkd.in/gQMFFu9n and to register:?https://lnkd.in/g4TbpZKC. We would love to have your support of this fun event. Funds received assist those affected by ALSP. Please help spread the word and share with your network. Thank you.?
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Our very own Samantha Charleston at the Global Genes Summit sharing her experience on how to activate your community and build connections and partnerships #weekinrare #kansascity
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Our Bloom Syndrome Grant Program LOI Deadline is fast approaching! Apply Today! Deadline: Friday, September 27 Our Bloom Syndrome Grant Program is seeking grant applications to increase the rate of discovery of the mechanisms of BSyn and, importantly, lead to treatments and/or cures for BSyn. While the RFA is intentionally broad in scope, priority will be given to grants that cover the following areas: 1. Improve knowledge of Bloom syndrome tumor biology. One 2-year grant for up to $150,000 (total cost). 2. Literature review, critical assessment & study proposal. Up to four one-year grants for up to $50,000 each (total cost). 3. Global cancer surveillance & novel cancer research in Bloom Syndrome. One 2-year grant for up to $150,000 (total cost). For more information and to apply follow the link: https://lnkd.in/g7TqKsbj
Bloom Syndrome Grant Program — Orphan Disease Center
orphandiseasecenter.med.upenn.edu
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??????The 2024 #MDBR Pilot Grant Program is now open and accepting Letters of Interest (LOIs)! Apply here today: bit.ly/MDBR2024RFA LOIs are due Sept. 20th. Grants are open to the international research community. #raredisease #rfa #pennmedmdbr2024 #rarediseasegrants
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Join ODC for a Research Ethics and Policy Series book talk with Amy Dockser Marcus from the The Wall Street Journal on 10/9/24. Discussing her book "We the Scientists: How the Rise of Patient-Led Research is Changing Medicine". This conversation is not to be missed. Register today! #raredisease #RareDiseaseResearch https://lnkd.in/ewJDjtAS...
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Register today for the exclusive screening of "The Zebra & The Bear", a documentary film about how a mother’s fierce determination to save her daughter from a devastating ultra-rare disease leads her on a journey to raise millions of dollars and drive the development of a pioneering gene-therapy treatment. Don't miss this opportunity to watch this inspiring movie & connect with others in the rare community on Friday, August 2nd in University City. Hosted as part of the International MSD Scientific & Family Conference. https://lnkd.in/emXcMcWT
Film Screening at the International MSD Scientific & Family Conference
eventbrite.com