LANCASTER, Pa. — On October 27, 2023, the third International Limb Girdle Muscular Dystrophy (LGMD) Conference will be hosted by The Speak Foundation at the Grand Hyatt Washington, DC. The inaugural International LGMD conference was held in Chicago in 2019 and over 400 patients, caregivers, physicians, researchers and advocacy groups...
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Wellington, New Zealand – The Malaghan Institute of Medical Research in collaboration with Wellington Zhaotai Therapies Limited today announced results of its phase 1 dose escalation trial of a new third generation anti-CD19 chimeric antigen receptor (CAR) T-cell therapy was presented at the American Society of Hematology (ASH) Annual Meeting...
Amsterdam, Netherlands – For more than 30 years, researchers have identified and quantified genetic and lifestyle factors for Alzheimer’s Disease (AD) and other dementias. Despite extensive research, the fundamental mechanisms behind these neurodegenerative diseases are still not fully understood, and effective management of AD is taking longer than expected. There...
David Fajgenbaum, MD, was a college football player at Georgetown University in 2004 when his mother passed away from cancer. The loss inspired the athlete to become a doctor himself: “I wanted to treat patients in memory of my mom,” he says. But three years into medical school, the then-25-year-old’s...
It is humbling to know that we are the beneficiaries of those who have gone before us. We stand on the shoulders of those who worked hard for us to benefit from their efforts and discoveries. We need not look further than the name of the disease that touches this...
ATLANTA — The first-ever national estimate among a nationally representative sample of U.S. children revealed that 3 out of every 1,000 children between the age of 6 and 17 in the United States have been diagnosed with Tourette Syndrome (TS), according to a study by the Centers for Disease Control...
MOBILE, Ala. — A special story about a three-year-old boy who needs the public’s help. Alijah Cunningham was born with a rare disease that caused him to lose all of his fingers and toes. Now, his parents are on a journey to gift him with what he lost. But you...
SAN FRANCISCO, Calif. – Thryv Therapeutics Inc., a clinical-stage biotechnology company advancing novel serum glucocorticoid inducible kinase 1 (SGK1) inhibitors for inherited cardiac arrhythmias, cardiometabolic diseases, and cardiomyopathies, today announced key development milestones for its THRV-1268 clinical program: patient dosing has commenced in the Phase 2/3 Wave II clinical study evaluating THRV-1268...
MONTREAL, Canada – Thryv Therapeutics Inc., a clinical-stage biotechnology company advancing novel serum glucocorticoid inducible kinase 1 (SGK1) inhibitors for inherited cardiac arrhythmias, cardiometabolic diseases, and cardiomyopathies, today announced the initiation of the Wave II clinical study, a pivotal Phase 2/3 clinical research study in adults diagnosed with Long QT Syndrome Type 2...
By Raffaele Pereno STOCKHOLM, Sweden – Reporting at BioEurope 2024, CheckOrphan had the opportunity to meet a promising biotech company, whose lead product seeks to stop the progression of amyotrophic lateral sclerosis (ALS) in its tracks. Tikomed AB is a privately owned pharmaceutical company based in Viken, Sweden. Adam Bruce,...