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Taysha Gene Therapies Joins the Rare Disease Company Coalition

Published: May 19, 2021

Joined an alliance of life science companies dedicated to rare disease therapy development to support advocacy for policies that will improve processes for timely and cost-effective delivery of treatments

DALLAS (BUSINESS WIRE) Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has joined the newly formed Rare Disease Company Coalition, a first-of-its-kind alignment of life sciences companies committed to discovering, developing and delivering rare disease treatments for the patients they collectively serve. ....

Aeglea Biotherapeutics , Acceleron Pharma , Alnylam Pharmaceuticals , Taysha Gene Therapies Inc , Rare Disease Company Coalition , Agios Pharmaceuticals , Orphazyme Us Inc , Ut Southwestern Gene Therapy Program , Gene Therapies , Chief Executive Officer , Rare Disease Company , Gene Therapy Program , தாய்ஷ்ா கீந் சிகிச்சைகள் இன்க் , உட் தென்மேற்கு கீந் சிகிச்சை ப்ரோக்ர்யாம் , கீந் சிகிச்சைகள் , தலைமை நிர்வாகி அதிகாரி , ரேர் நோய் நிறுவனம் , கீந் சிகிச்சை ப்ரோக்ர்யாம் ,

Detecting rare disease: Revealing the methods, motivations, and implications

Washington, DC
Dr. Summar is well-known for his pioneering work in caring for children diagnosed with rare diseases. He came to Children’s National Hospital in Washington, DC, in 2010 from Vanderbilt University and holds board certifications in both pediatrics and clinical genetics as well as biochemical genetics. At Children’s National, he leads the Division of Genetics and Metabolism, currently the largest clinical division of its kind in the world, overseeing 8,000 patients a year with rare diseases. His laboratory works on both devices and treatments for patients with genetic diseases while also supporting advancements in mainstream medicine through education about these disorders. His work has resulted in new drugs in U.S. Food and Drug Administration trials for patients with congenital heart disease and premature birth. Dr. Summar holds more than 60 patents and has published more than 160 peer-reviewed research studies. He developed and launched the world’s first R ....

United States , Hong Kong , United Kingdom , University Of Cape Town , Western Cape , South Africa , Eteläuomen Läi , University Of Illinois , Georgetown University , District Of Columbia , University Of Texas , Jimeng Sun , Sean Sanders , Cynthia Tifft , University Of Cambridge , Children National Research Innovation Campus , College Of Computing , Institute Medical Genetics Training Program , National Institutes Of Health , Drug Administration , Georgia Institute Of Technology , Rare Disease Institute , Finnish Institute For Health , Health Innovation Professor , Children National Hospital , National Human Genome Research Institute ,

The Children's National Hospital Rare Disease Institute And Takeda Partner To Standardize Care For Patients With Rare Diseases

The Children s National Hospital Rare Disease Institute And Takeda Partner To Standardize Care For Patients With Rare Diseases
A five-year commitment from Takeda will help Children s National launch a first-of-its-kind rare disease clinical protocol program.
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WASHINGTON and CAMBRIDGE, Mass., March 1, 2021 /PRNewswire/ Children s National Hospital and Takeda Pharmaceutical Company Limited
(TSE:4502/NYSE:TAK) ( Takeda ) today announced the creation of the Rare Disease Clinical Activity Protocols (Rare-CAP) program, which will establish a networked system for the development, dissemination and curation of protocols to help standardize the process of diagnosis and care for patients with rare diseases. ....

Walter Reed Army Medical Center , District Of Columbia , United States , National Institutes Of Health , United Kingdom , Tom Koutsavlis , Children National , Takeda Pharmaceutical Company Limited , Rare Diseases Information Center , Takeda Pharmaceutical Company , Children National Research Innovation Campus , Children National Research Institute , Us Medical Affairs , Sheikh Zayed Institute , Children National Rare Disease Institute , World Health Organization , Rare Disease Institute , Prnewswire Children National Hospital , Division Of Genetics , Children National Hospital , Clinical Center , National Hospital , Rare Disease Clinical Activity Protocols , National Research , Innovation Campus , Walter Reed Army Medical ,

Rare Disease Diversity Coalition Formed To End Racial Disparities In Rare Disease Diagnosis, Research And Treatment

Rare Disease Diversity Coalition Formed To End Racial Disparities In Rare Disease Diagnosis, Research And Treatment
NBA Champion Alonzo Mourning, White House COVID Health Equity Task Force Chair Dr. Marcella Nunez-Smith, Rep. G. K. Butterfield and Leading Rare Disease Experts to Join RDDC on February 23rd for Launch of Diversity Coalition
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WASHINGTON, Feb. 23, 2021 /PRNewswire/ Today, the Black Women s Health Imperative (BWHI) hosts the first public meeting of the Rare Disease Diversity Coalition (RDDC). RDDC seeks to address the pressing challenges faced by marginalized populations and identify potential solutions. Led by BWHI and comprised of a diverse group of healthcare organizations, patient advocacy groups, and industry experts, the RDDC is poised to achieve action in the years to come by: ....

United States , The White House , District Of Columbia , Deric Dube , Beverley Francis Gibson , Laura Weidner , Donna Cryer , Brian Thompson , Saira Sultan , Tammy Boyd , Cassandra Mccullough , Matthewl Watley , Selena Rios , Kim Smith Whitley , Tamar Thompson , Yousra Yusuf , Debbie Drell , Anthonyj Brownlow , Lauren Lee , Linda Goler Blount , Kimberly Haugstad , Amy Hinojosa , Ashley John , Courtney Pieczynski Keplinger , John Burns , Julietk Choi ,

Rare Disease Diversity Coalition Formed To End Racial Disparities in Rare Disease Diagnosis, Research and Treatment

Rare Disease Diversity Coalition Formed To End Racial Disparities in Rare Disease Diagnosis, Research and Treatment
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United States , The White House , District Of Columbia , Deric Dube , Laura Weidner , Donna Cryer , Brian Thompson , Saira Sultan , Tammy Boyd , Cassandra Mccullough , Matthewl Watley , Lesli Foster , Selena Rios , Kim Smith Whitley , Tamar Thompson , Yousra Yusuf , Debbie Drell , Anthonyj Brownlow , Lauren Lee , Linda Goler Blount , Kimberly Haugstad , Amy Hinojosa , Ashley John , Courtney Pieczynski Keplinger , John Burns , Julietk Choi ,