Rare Disease Research

Selected news for the healthcare topic - Rare Disease Research, collected since 10/2017. There are 268 items in the archive. This healthcare topic shares news with Rare Diseases, Pfizer, Patient Advocacy and many others.

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

Selected Headlines

Date Headline (link) Source Relevant Snippet
3/14/2022 Rare diseases, common challenges Nature The genetics community has a particularly important part to play in accelerating rare disease research and contributing to improving diagnosis and treatment. Innovations in sequencing technology and machine learning approaches have positively affected diagnostic success, but more coordinated efforts are needed to move towards effective therapies or even cures for these important, and sometimes overlooked, class of diseases.Rare Disease Day was recently held on 28 February 2022, which aimed ...
3/10/2022 Emmes Acquires Casimir, Its Fourth Major Acquisition PR Newswire ... of disease progression and treatment benefit. Terms of the transaction were not disclosed.Emmes' acquisition of Casimir – the fourth since Behrman's investment in 2019 – will further accelerate the Company's strategic focus on rare disease research , building on Emmes' successful acquisition of Orphan Reach last year. With the added enrichment of Casimir's proprietary clinical outcomes technology, Emmes will be even better positioned as the industry-leading CRO in rare ...
3/10/2022 Has COVID-19 transformed the future of rare disease research? illingworthresearch.com Has COVID-19 transformed the future of rare disease research?Figures published in Tufts CSDD Impact Reports in 20201 highlight the passion within the rare disease population to be involved in research, with dropout rates lower than in any other indication. Recruitment rates were higher across all indications than in previous years. The average dropout rate of 19.1 per cent across all indications demonstrates how incredibly low the rare disease ...
3/7/2022 KCNT1 Epilepsy Foundation Launches Digital Natural History Study in Partnership with Invitae Ciitizen PRWeb ... accurately understanding the disease. It aims to augment traditional studies by amassing retrospective data that ultimately reduces the time needed for research and drug development.“We are proud to be at the forefront of rare disease research and collaborate with Invitae to redefine the drug development process for KCNT1,” said Justin West, MD, co-founder and Director of Clinical Medicine for the KCNT1 Epilepsy Foundation. “These analytics are expected ...
3/7/2022 A call for an integrated approach to improve efficiency, equity and sustainability in rare disease research in the United States Nature To build a more efficient, equitable and sustainable approach to rare disease research in the United States, we must prioritize integrated research infrastructure and approaches that focus on understanding connections across rare diseases.This is a preview of subscription contentAccess optionsSubscribe to JournalGet full journal access for 1 year55,14 €only 4,60 € per issueAll prices are NET prices.VAT will be added later in ...
2/28/2022 Rare disease research on ScienceOpen scienceopen.com Rare disease research onRare Disease Day, observed annually on February 28th, is a globally coordinated movement aimed at raising awareness about rare diseases and the consequences they have for the 300 million people who suffer from them worldwide.Rare diseases affect not only the people who have them but also their families and caregivers. They are also sometimes known as orphan diseases because drug companies have a low interest ...
2/28/2022 Alltrna Appoints Rare Disease Clinical Research Expert, Dr. Anne Pariser, as Vice President, Medical and Regulatory Affairs PR Newswire ... she founded the Rare Diseases Program and predominantly focused on rare diseases drug review."Anne has been an exceptional force in government organizations, first at the FDA and then at the NIH, to propel rare disease research , translational science, and drug development," said Lovisa Afzelius , Ph.D., MBA, Origination Partner at Flagship Pioneering and Founding CEO of Alltrna. "We are thrilled she has joined our team to lead Alltrna's strategy for ...
2/23/2022 New Report Details State of Rare Disease R&D, Investment and Treatment | Business Wire Business Wire ... leading global rare disease stakeholders outlining advancements in rare diseases and what comes next.An aggregate of rare disease trends, highlights and commentary from leaders in the industry, the report outlines progress made in rare disease research , diagnosis, development, regulation, funding and treatment, introducing new and unique data. Key findings within the report include:There are as many as 10,869 rare diseases worldwide, as opposed to the often used 7,000 ...
2/23/2022 New Report Details State of Rare Disease R&D, Investment and Treatment Business Wire ... leading global rare disease stakeholders outlining advancements in rare diseases and what comes next.An aggregate of rare disease trends, highlights and commentary from leaders in the industry, the report outlines progress made in rare disease research , diagnosis, development, regulation, funding and treatment, introducing new and unique data. Key findings within the report include:- There are as many as 10,869 rare diseases worldwide, as opposed to the often used 7,000 ...
2/14/2022 CSL Behring Partners with Uplifting Athletes for Rare Disease Research and Honors Those Making an Impact montco.today CSL Behring Partners with Uplifting Athletes for Rare Disease Research and Honors Those Making an ImpactAt the celebratory event, presented by global biotherapies leader CSL Behring , Uplifting Athletes awarded $180,000 in research grants to nine promising young investigative rare disease researchers and honored the six newest members of the organization’s Rare Disease Championship Team―an exemplary group of college football players who support Uplifting Athletes’ mission to ...
2/7/2022 From the Athletic Field to the Research Lab ― How Uplifting Athletes is Supporting the Rare Disease Community tncontentexchange.com ... Draft on February 5, 2022, at Lincoln Financial Field in Philadelphia. At the celebratory event, presented by global biotherapies leader CSL Behring , Uplifting Athletes awarded $180,000 in research grants to nine promising young investigative rare disease research ers, and honored the six newest members of the organization's Rare Disease Championship Team―an exemplary group of college football players who support Uplifting Athletes' mission to inspire the Rare Disease Community with hope ...
2/7/2022 From the Athletic Field to the Research Lab ― How Uplifting Athletes is Supporting the Rare Disease Community | State bradfordera.com ... Draft on February 5, 2022 , at Lincoln Financial Field in Philadelphia . At the celebratory event, presented by global biotherapies leader CSL Behring , Uplifting Athletes awarded $180,000 in research grants to nine promising young investigative rare disease research ers, and honored the six newest members of the organization's Rare Disease Championship Team―an exemplary group of college football players who support Uplifting Athletes' mission to inspire the Rare Disease Community with hope ...
2/7/2022 From the Athletic Field to the Research Lab ― How Uplifting Athletes is Supporting the Rare Disease Community PRWeb ... Draft on February 5, 2022, at Lincoln Financial Field in Philadelphia. At the celebratory event, presented by global biotherapies leader CSL Behring , Uplifting Athletes awarded $180,000 in research grants to nine promising young investigative rare disease research ers, and honored the six newest members of the organization’s Rare Disease Championship Team―an exemplary group of college football players who support Uplifting Athletes’ mission to inspire the Rare Disease Community ...
1/10/2022 Professor Damian Smedley discusses computational biology in rare disease research QMUL biology research raredisea... | News Medical | Scoopnest scoopnest.com Professor Damian Smedley discusses computational biology in rare disease research biology raredisease genomics sequencing a href='/s/wholegenomesequencing' alt='wholegenomesequencing' /' wholegenomesequencing /a /p ...
12/9/2021 CANbridge Signs Collaboration Agreement with the Peking Union Medical College on Rare Disease Research | Business Wire Business Wire ... Mass.--( BUSINESS WIRE )--CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that it signed the collaboration on a rare disease research agreement with the Peking Union Medical College Hospital (PUMCH), on December 9, 2021. Under terms of the agreement, the two parties will collaborate on research and development in rare disease drug innovation, translational ...
12/9/2021 CANbridge Signs Collaboration Agreement with the Peking Union Medical College on Rare Disease Research Business Wire ... Dec 9, 2021--CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that it signed the collaboration on a rare disease research agreement with the Peking Union Medical College Hospital (PUMCH), on December 9, 2021. Under terms of the agreement, the two parties will collaborate on research and development in rare disease drug innovation, translational ...
9/22/2021 Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness pompediseasenews.com Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation — are organizing fundraisers to help young researchers find treatments for the ...
9/22/2021 Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness rettsyndromenews.com Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation — are organizing fundraisers to help young researchers find treatments for the ...
8/25/2021 Morning Headlines 8/25/21 HISTalk ... Raises $20 Million of Growth Equity from Heritage Group Acute care telehealth and teleICU service provider Equum Medical raises $20 million in growth equity. AllStripes Announces $50 Million Series B Financing to Advance Global Rare Disease Research AllStripes, which offers real-world and patient data to support rare disease research , raises $50 million in a Series B funding round ...
8/24/2021 AllStripes Announces $50 Million Series B Financing to Advance Global Rare Disease Research Yahoo News Lux Capital Led Funding, Joined by JAZZ Venture PartnersFinancing to Advance AllStripes’ Capabilities and Launch New Rare Disease Research ProgramsSAN FRANCISCO, August 24, 2021 --( BUSINESS WIRE )-- AllStripes ("Company"), a healthcare technology company dedicated to unlocking treatments for people affected by rare diseases, today announced the Company completed a $50 million Series B financing round.This press release features multimedia. View the full release here: https://www.businesswire.com/news ...