DALLAS, TX — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced positive top-line results after the completion of the 2-year Phase 2b RESTORE randomized, controlled clinical trial of its lead program, MCO-010, a mutation-agnostic gene therapy...
treatment News
Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization (WHO) has selected "sonpiretigene isteparvovec" for the nonproprietary name of the Company's MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP) and Stargardt disease.
DALLAS, Texas — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today provided an update following recent regulatory discussions with the U.S. Food and Drug Administration (FDA) and from the Scientific Advice meeting with the Icelandic Medicines Agency (IMA),...
Philadelphia, PA – Thursday afternoon marked a major regulatory milestone. In one of the year’s most highly anticipated FDA decisions, the regulator approved Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) as the first-ever therapy for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (NASH). Or was the approval for metabolic dysfunction-associated steatohepatitis (MASH)? If...
DUBLIN – Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the National Comprehensive Cancer Network® (NCCN) added Rylaze™ (asparaginase erwinia chrysanthemi (recombinant)-rywn) to the Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Lymphoblastic Leukemia (ALL), for both pediatric and adult patients. The NCCN Guidelines for ALL and the NCCN Guidelines for Pediatric...
OXFORD, England – Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the National Institute for Health and Care Excellence (NICE) has issued a positive Final Appraisal Document (FAD) recommending Sunosi®▼ (solriamfetol) for adults with excessive daytime sleepiness (EDS) caused by narcolepsy.[1] Solriamfetol is recommended as an option for treating EDS in...
LAS VEGAS — Bone sarcoma is a rare and aggressive form of cancer that originates in the bone tissue. It poses significant challenges during its treatment due to several factors. Firstly, its rarity often leads to delayed diagnosis, as symptoms can mimic other common conditions. Additionally, bone sarcoma typically affects...
Bethesda, Maryland – Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented by a single normal copy of a defective gene. This means the potential exists that scientists could one day tailor a gene therapy to treat the disease. They also...
PLYMOUTH MEETING, PA — The National Comprehensive Cancer Network® (NCCN®) is announcing several new updates to the cancer treatment practices listed in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®). The evidence-based expert consensus recommendations are the most frequently updated guidelines for any area of medicine and are used...
Boston, Mass. – An important discovery from the NCI-MATCH precision medicine initiative is being published today in Clinical Cancer Research, a journal of the American Association for Cancer Research. Trastuzumab-pertuzumab, a drug combination approved by the US Food and Drug Administration (FDA) to treat patients with human epidermal growth factor...