MIAMI, Florida and VANCOUVER, British Columbia — RESTEM, a clinical-stage biotechnology company that develops off-the-shelf, next-generation cell therapies designed to modulate the immune system, today announced the initiation of a Phase 1/2a study of Restem-L, the Company’s umbilical lining modified progenitor cells (UMPCs) off-the-shelf platform, for the treatment of facioscapulohumeral...
treatment News
SOUTH PLAINFIELD, N.J., – PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented pooled placebo data from multiple studies in which the results confirmed the clinical benefit of EMFLAZA® (deflazacort) over prednisone for the treatment of Duchenne muscular dystrophy (DMD). The results demonstrated that DMD patients on daily EMFLAZA performed better on...
The REAL8 trial showed that after 52 weeks, once-weekly Sogroya® (somapacitan) had similar clinical outcomes and safety profile to once-daily Norditropin® (somatropin) in children born small for gestational age (SGA)1, or with Noonan syndrome (NS)2, or with idiopathic short stature3. Superiority was achieved for once-weekly somapacitan versus daily growth hormone in children...
JINAN, China — The results of the phase II study on Qilu Pharmaceutical’s novel anticancer drug iparomlimab and tuvonralimab (QL1706) were published online in Signal Transduction and Targeted Therapy (Impact Factor=39.3). The study investigated the use of QL1706 in combination with chemotherapy, with or without bevacizumab, for the treatment of...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented a new analysis of five-year results that shows its novel gene therapy, PTC-AADC, leads to profound improvements in children with aromatic L-Amino acid decarboxylase (AADC) deficiency, a previously intractable, fatal and devastating rare disorder of the central nervous system....
BERKELEY, Calif. — ResVita Bio, a therapeutics company focused on treatments for skin diseases, today announced that the FDA awarded the Orphan Drug Designation to RVB-003 for the treatment of Netherton Syndrome, a chronic and life-threatening skin disorder. This milestone, following the FDAs previous granting of the Rare Pediatric Disease...
PETALUMA, Calif. — RetinalGeniX™ Technologies Inc. today announced that it has contracted with MEDsan, Inc. to provide diagnostic testing services for its Institutional Review Board (IRB) to conduct a study to personalize medical evaluations for patients receiving treatment for wet macular degeneration. “We are pleased to extend our services into...
Houston, TX – Texas Children’s Hospital, an internationally-recognized, top-ranked children’s hospital and pediatric research center affiliated with Baylor College of Medicine, is the first to deliver a novel gene therapy to treat Rett syndrome in pediatric patients. Two female patients with Rett syndrome were the first children worldwide to receive...
NASHVILLE, Tenn. – Revance Therapeutics, Inc. (Nasdaq: RVNC), a biotechnology company focused on innovative aesthetic and therapeutic offerings, today announced that Shanghai Fosun Pharmaceutical Industrial Development Co., Ltd., (Fosun Pharma Industrial), a wholly-owned subsidiary of Shanghai Fosun Pharmaceutical (Group) Co., Ltd, (Fosun Pharma), a leading healthcare group in China, has...
MIAMI, FL – Ludwig Enterprises, Inc. (the “Company”), a biotech innovator in mRNA diagnostics and AI-driven health solutions, announced today the initial introduction of its lead product, Revealia Breast™, a non-invasive breast cancer screening test currently in advanced development. This innovative diagnostic tool uses a simple cheek swab to detect cancer-related...