Industry Archives - Page 2 of 12 - NORD (National Organization for Rare Disorders)

February 23, 2021

TOPIC: Press Releases, Featured News, Patients & Members, Advocacy, Industry

NORD Announces This Year’s Heroes of Rare Disease: The 2021 Rare Impact Award Honorees

Posted at February 2, 2021 10:17 am by Valaree DonFrancesco

Washington, DC, February 23, 2021 — Today the National Organization for Rare Disorders (NORD®) announced this year’s Rare Impact Award honorees. These outstanding individuals, organizations and industry innovators will be honored for their exceptional work benefiting the rare disease community in a virtual event streaming on June 28, 2021 at 7:00pm ET. The Rare Impact Awards program is part… Read More

Tags: Rare Impact Awards, Desmoid Tumor Research Foundation, Peter Marks, Public Health Leadership Award, Abbey S. Meyers Leadership Award, MD, Living Rare Living Stronger Patient and Family Form, Living Rare, Loxo Oncology, Incyte, alnylam pharmaceuticals, Horizon Therapeutics, Blueprint Medicines, Jade Day, Daniela Delgado, Robert Long, Kam Redlawsk, Susan Fernback, Cathleen Lutz, Natasha Shur, Jason Sicklick, Danyelle Sun, Mary Wootten, Hannah Kane, State Representative from Massachusetts, Patty Murray, Senator from Washington, Oxlumo, AstraZeneca, Koselugo, Ayvakit, Eiger Biopharmaceuticals, Zokinvy, Tepezza, Pemazyre, Eli Lilly, Retevmo, Regeneron, Inmazeb, Ridgeback Biotherapeutics, Ebanga

December 16, 2020

TOPIC: Featured News, Research, Industry, Students for Rare

NORD Students for Rare Member Provides Her Perspective on “Unlocking the Power of Health Care Data” Summit Panel

Posted at December 12, 2020 08:38 am by Jennifer Ryan

As a 2020 scholarship recipient, I was able to attend the NORD Rare Diseases and Orphan Products and Breakthrough Summit in October and to hear from inspiring patients, hard-working medical professionals and research experts. Specifically, I was very drawn to the “Unlocking the Power of Health Care Data” panel, which discussed how data-driven health care is being used… Read More

Tags: NORD Summit, Rare Diseases and Orphan Products Breakthrough Summit, Mitochondrial Disease, Rare Disease Cures Accelerator Data and Analytics Platform, RDCA-DAP, Eversana, Critical Path Institute, Jane Larkindale, Kira Mann, MitoAction Mobile

November 2, 2020

TOPIC: Featured News, Patients & Members, Research, Advocacy, Industry

NORD and Patient Advocacy Groups Host an EL-PFDD Meeting on Krabbe Disease

Posted at November 11, 2020 02:25 pm by Debbie Drell

One of the most important gatherings of the Krabbe disease community occurred in collaboration with the US Food and Drug Administration (FDA) on October 29, and it was 100% virtual with live, interactive components. FDA regulators, academic researchers and industry drug developers came together with Krabbe disease patients, caregiversRead More

Tags: Krabbe disease, Voice of the Patient, KrabbeConnect, Partners for Krabbe Research, galactocerebrosidase, The Legacy of Angels Foundation, Hunter's Hope, Gain Therapeutics, Magenta Therapeutics, PassageBio, Neurogene, lysosomal storage disorder

October 21, 2020

TOPIC: Press Releases, Featured News, Patients & Members, Research, Advocacy, Industry, RDCA-DAP

RDCA-DAP First Year Milestones Highlighted at Virtual Workshop

Posted at October 10, 2020 11:14 am by Valaree DonFrancesco

TUCSON, Ariz. and WASHINGTON, D.C. October 21, 2020 — The Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD®) hosted the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) 2020 Virtual Workshop on Monday, October 19. The platform, funded by a cooperative agreement through the U.S. Food and Drug Administration (FDA), serves as… Read More

Tags: NORD, Peter L. Saltonstall, FDA, National Organization for Rare Disorders, Vanessa Boulanger, Inc., US Food and Drug Administration, Rare Disease Cures Accelerator Data and Analytics Platform, RDCA-DAP, Theresa Mullin, Critical Path Institute, Billy Dunn, Jane Larkindale, Michelle Campbell, Associate Director for Strategic Initiatives, Center for Drug Evaluation and Research (CDER), Klaus Romero, Otsuka America Pharmaceutical, Pravin Jadhav, Ph.D., M.S.c.

October 14, 2020

TOPIC: Featured News, Patients & Members, Research, Industry, RDCA-DAP

NORD and C-Path Release New Video, “RDCA-DAP: Shortening the Timeline for Developing New Treatments for Rare Diseases”

Posted at October 10, 2020 08:01 am by Valaree DonFrancesco

NORD and the Critical Path Institute (C-Path) are collaborating on a new initiative, the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP), bringing together data from different sourcesRead More

Tags: The Critical Path Institute, Rare Disease Cures Accelerator Data and Analytics Platform, RDCA-DAP, C-Path, drug development process