Rare Disease Research

Selected news for the healthcare topic - Rare Disease Research, collected since 10/2017. Recent stories appear in pharmamanufacturing.com and Business Wire. This healthcare topic shares news with Rare Diseases, Pfizer, Seng Cheng and many others.

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Healthcare Topics Rare Diseases, Biotech, Gene Therapy, Clinical Data, Medical Research, Clinical Research, Patient Community, National Institutes of Health, Food and Drug Administration, NIH, Orphan Drugs, Patient Registry
Companies Pfizer, Sangamo Therapeutics, Therachon
People Seng Cheng

Selected Headlines

Date Headline (link) Source Relevant Snippet
6/21/2021 Rare disease research: A powerful drop in the ocean pharmamanufacturing.com Rare disease research: A powerful drop in the ocean When niche research makes big waves In the 1960s, the average person did not give much thought to the comings and goings of horseshoe crabs. For decades prior, humans had been mindlessly grinding up the 10-legged creatures for use as fertilizer; but even that practice began to subside with the invention of artificial means. Hence the harmless sea dinosaurs were ...
6/15/2021 AllStripes Awarded as Technology Pioneer by World Economic Forum | Business Wire Business Wire ... rare diseases, was selected from hundreds of candidates as one of the World Economic Forum’s “Technology Pioneers” of 2021. AllStripes has developed a technology platform that generates FDA-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data, all at no cost to ...
6/15/2021 malvern-online.com ... rare diseases, was selected from hundreds of candidates as one of the World Economic Forum’s “Technology Pioneers” of 2021. AllStripes has developed a technology platform that generates FDA-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data, all at no cost to ...
6/15/2021 AllStripes Awarded as Technology Pioneer by World Economic Forum | Financial Buzz financialbuzz.com ... rare diseases, was selected from hundreds of candidates as one of the World Economic Forum’s “Technology Pioneers” of 2021. AllStripes has developed a technology platform that generates FDA-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data, all at no cost to ...
5/21/2021 Upcoming HealthVerity Event on RWD in Rare Disease Drug Development Pipeline healtheconomics.com Upcoming HealthVerity Event on RWD in Rare Disease Drug Development Pipeline Upcoming HealthVerity Event on RWD in Rare Disease Drug Development PipelineUsing in rare disease research offers a method to circumvent many of the traditional challenges facing researchers. HealthVerity will hold a live event on May 27 th at 12:00 PM EDT featuring insight on RWD sources and select cases where RWD was used to draw important conclusions.“In ...
5/21/2021 AmerisourceBergen Interview Highlights the Power of RWE in Drug Development Pipeline healtheconomics.com ... experts focused on the role of real world evidence (RWE) in the drug development pipeline. In addition to improving the quality of conclusions drawn from clinical trials, researchers are increasingly turning to RWE in rare disease research .Xcenda’s Vice President of Scientific Consulting Michael Eaddy remarks, “[F]or orphan diseases of the sample size of populations are just so small that randomization could be problematic and require for ...
5/14/2021 A Cat who Curates Rare Mice globalgenes.org ... Orphan Disease Center today features more than 8,500 unique strains including more than 1,700 live colonies that are distributed to the scientific community. We spoke to Lutz about the role mouse models play in rare disease research , how new gene editing technologies are changing the development of mouse models, and why new technologies are unlikely to displace their use anytime soon. Daniel Levine: Thanks for joining us. Cat Lutz: Thanks ...
5/12/2021 Optum Whitepaper on RWD in Rare Disease Research healtheconomics.com Optum Whitepaper on RWD in Rare Disease Research Optum Whitepaper on RWD in Rare Disease ResearchOptum is offering a whitepaper that provides insight into how real world data (RWD) can accelerate rare disease research. The paper identifies deidentified patient data, including electronic health records, as a robust source of RWD.“Rare disease research and treatment development is uniquely challenging given the limited understanding of rare and genetic diseases. De ...
5/12/2021 Tennessee Rare Disease Advisory Council to Inform TennCARE on Pharmaceutical Treatments - Nashville Medical News nashvillemedicalnews.com ... elected at its inaugural meeting its chairman Dr. Scott Strome, Executive Dean of the University of Tennessee College of Medicine and vice chancellor for clinical affairs at UT Health Science Center. Dr. Strome brings rare disease research and clinical experience to the TN-RDAC.The legislation that enabled Tennessee's RDAC was sponsored by Sen. Shane Reeves (R-Murfreesboro) and Rep. David Hawk (R- Greeneville) and was signed into law by ...
5/10/2021 Validating online approaches for rare disease research using latent class mixture modeling biomedcentral.com Research - Open Access - Published: Validating online approaches for rare disease research using latent class mixture modeling 209 (2021) Abstract Background Rare disease patients are geographically dispersed, posing challenges to research. Some researchers have partnered with patient organizations and used web-based approaches to overcome geographic recruitment barriers. Critics of such methods claim that samples are homogenous and do not represent the broader patient population—as patients recruited from patient organizations ...
5/7/2021 CANbridge Acquires Rights to Gene Therapy Candidates fabrydiseasenews.com ... Kong, and Macau).“This important agreement advances our three-prong strategy to build CANbridge’s next-generation rare disease treatment pipeline: in-house research capability, as supported by the recent opening of our CANbridge rare disease research facility, in Massachusetts; collaborative academic research agreements, such as the two we have with the Horae Gene Therapy Center, at the University of Massachusetts Medical School; and partnerships with innovative biotechnology companies,” ...
5/4/2021 Soleno Therapeutics Announces Presentation of Positive Behavioral Data from Ongoing Extension Study of DCCR for Treatment of Prader-Willi Syndrome Globe Newswire ... PWS, interviews are being conducted with caregivers of C602 study participants to characterize individual patient experiences with DCCR. The interviews are performed at multiple timepoints during the Phase 3 program by Casimir Inc., a rare disease research organization. The poster highlights the analysis of a subset of 48 interviews of caregivers whose child had received at least 13 weeks of DCCR treatment in C602. Folia Health, which utilizes a data ...
5/4/2021 Soleno Therapeutics Announces Presentation of Positive Behavioral Data from Ongoing Extension Study of DCCR for Treatment of Prader-Willi Syndrome roboticulized.com ... PWS, interviews are being conducted with caregivers of C602 study participants to characterize individual patient experiences with DCCR. The interviews are performed at multiple timepoints during the Phase 3 program by Casimir Inc., a rare disease research organization. The poster highlights the analysis of a subset of 48 interviews of caregivers whose child had received at least 13 weeks of DCCR treatment in C602. Folia Health, which utilizes a data ...
4/27/2021 Global Data Standards Being Developed for Rare Disease Research bionewsfeeds.com Global Data Standards Being Developed for Rare Disease Research https://bionewsfeeds.com/2021/04/27/global-data-standards-being-developed-for-rare-disease-research/Global data standards for conducting research on rare diseases, such as Lambert-Eaton myasthenic syndrome (LEMS), are being developed by a new partnership between the Clinical Data Interchange Standards Consortium (CDISC) and the National Organization for Rare Disorders (NORD). Rare disorders in the U.S. are defined as those affecting ...
4/26/2021 WASHINGTON Latino News washingtonlatinonews.com ... faced by communities of color, with a special emphasis on the inequities that rare disease patients of color experience. The group will discuss its efforts to increase the participation of communities of color in rare disease research and clinical trials by addressing regulatory flexibility.The meeting will discuss the coalition's ongoing work to implement the priorities outlined in its 2021 " Charting the Path Forward for Equity in Rare Diseases " report ...
4/21/2021 SMRT Sequencing Detects Clinically Significant Repeat Changes In Triplet Expansion Disorders pacb.com ... When size matters and you need to be able to detect both single nucleotide changes as well as large repeated sequences, SMRT Sequencing on the Sequel II System is the way to go, concluded rare disease research ers at Centre de Recherche en Myologie at Sorbonne Université/INSERMStéphanie Tomé ( @TomeStephanie ) and colleagues used the highly sensitive, comprehensive long-read sequencing to investigate myotonic dystrophy type 1 (DM1), the most ...
3/31/2021 Uplifting Athletes Presents $140,000 in Rare Disease Research Grants at 2021 Young Investigator Draft mysanantonio.com Uplifting Athletes Presents $140,000 in Rare Disease Research Grants at 2021 Young Investigator Draft PRWebSports and Science Team Up to Honor Emerging Medical Researchers at Annual Celebration Modeled After NFL Draft PHILADELPHIAUplifting Athletes , a nonprofit organization serving the Rare Disease Community, awarded $140,000 in medical research grants last night at its Young Investigator Draft Presented by CSL Behring . Seven promising young researchers received unrestricted $20,000 grants to support ...
3/25/2021 Second Phase 3 Study of Pamrevlumab Recruiting Boys With DMD bionewsfeeds.com ... muscular dystrophy (DMD) who are able to walk. The trial, LELANTOS-2 (NCT04632940), is enrolling approximately 70 participants ages 6 to 12 at three U.S. sites — Arkansas Children’s Hospital in Little Rock, Rare Disease Research Center in Atlanta, Georgia, and University of Massachusetts Memorial Medical Center in Worcester. “The initiation of LELANTOS-2, the second Phase 3 clinical†...
3/1/2021 Focus on Patients to Boost Rare Disease Research Success worldwide.com Focus on Patients to Boost Rare Disease Research Success Focus on Patients to Boost Rare Disease Research Success By The Editors of Uncommon Conversations, March 1, 2021Far too often, CROs plan a rare disease trial and then go looking for patients to recruit. At Worldwide, we believe the best approach to rare disease research is the other way around: First, find your patients. Talk to them. Understand their experience ...
2/26/2021 Rare Disease Day and Licensing Opportunities National Institutes of Health ... and the public. It is observed in 103 countries with thousands of events occurring. On March 1st, NCATS and the NIH Clinical Center will host their Rare Disease Day conference to highlight NIH-supported rare disease research and development, share the latest rare disease information, and share patients’ stories. The event will feature a myriad of speakers such as the NIH Director, patients and their families, and rare disease ...