Rare and Genetic Disease Network
Featured
KEYNOTE-890: TAVO in Combination with Keytruda for Late-Stage Triple Negative Breast Cancer Patients
Dan O’Connor, President, Director and Chief Executive Officer of OncoSec discusses the KEYNOTE-890 study, a Phase 2 trial for the treatment of late-stage triple negative breast cancer (TNBC) with TAVO (intratumoral plasma encoded IL-12, or tavokinogene telseplasmid, plus electroporation) in combination with Merck’s Keytruda (pembrolizumab).
Sanford Health Overview and It’s Research in Rare Diseases
Benjamin Forred, Translational Research Project Manager at Sanford Health in Sioux Falls, South Dakota, discusses his organization which is one of the largest health systems in the United States, including research into rare diseases.
KEYNOTE-695 Study of TAVO + Keytruda in Refractory Metastatic Melanoma
Dan O’Connor, President, Director and Chief Executive Officer of OncoSec discusses the KEYNOTE-695 Study of TAVO + Keytruda in Refractory Metastatic Melanoma.
Metachromatic Leukodystrophy and the MLD Foundation
Dean Suhr, President of the MLD Foundation, provides an overview of Metachromatic leukodystrophy (MLD) and an overview of his advoacy organization.
Gene Testing for Inherited Eye Diseases
Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses gene testing for inherited eye diseases, including his company’s initative EyeWant2Know.com.
Jen McNary Discusses Rare Disease Week and the Role of Advocates
Jennifer McNary is a consultant, speaker, and rare disease advocate. She is also a mother of two boys with Duchenne muscular dystrophy. In this video, Jennifer discusses Rare Disease Day and her evolving role of Advocate to Industry consultant.
NIH Director Francis Collins Talks About Rare Disease Day
Dr. Collins began his talk by acknowledging that Rare Diseases are more relevant that ever with all of NIH’s 27 institutes and centers having some investment in rare disease research.
The Role of IL-12 in Anti-PD-1 Therapies with TAVO
Dan O’Connor, President, Director and Chief Executive Officer of OncoSec discusses his company and it’s focus on developing cytokine-based intratumoral immunotherapies to stimulate the body’s immune system to target and attack cancer.
Alnylam Overview and it’s Focus on RNA Interference
Pushkal Garg, MD, Chief Medical Officer at Alnylam provides an overview of his company and its focus on RNA interference (RNAi).
ZYN002: Being Evaluated for Fragile X Syndrome
Armando Anido, Chief Executive Officer at Zynerba Pharmaceuticals discusses his company’s lead product candidate ZYN002, a CBD gel currently being evaluated for Fragile X syndrome.
Michael E. Shy, MD: Natural History, Biomarkers, and Gene Identification
Michael E. Shy, MD, Director, Division of Neuromuscular Medicine, Neurology at the University of Iowa Health Care, discusses Natural History, Biomarkers, and Gene Identification during the Rare Disease Day at NIH 2019 on Thursday, February 28, 2019.
James C. Greenwood, President of the Biotechnology Innovation Organization (BIO)
James C. Greenwood is President and CEO of the Biotechnology Innovation Organization (BIO) in Washington, DC. BIO represents 1,000 biotechnology companies, academic institutions, state biotechnology centers, and related organizations across the United States and in more than 30 countries worldwide.
Special Reports
Rising to the Challenge: Developing New Medicines for Rare Disease
Rare in Common Podcast
American Society of Hematology Conference Coverage
Considering Rare Diseases for Challenging Cases
Duchenne Muscular Dystrophy
Rare Disease Diagnosis: Huntington’s Disease
Rising to the Challenge: Developing New Medicines for Rare Disease
Rare in Common Podcast
American Society of Hematology Conference Coverage
Considering Rare Diseases for Challenging Cases
Duchenne Muscular Dystrophy
