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....
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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...
Using data generated from patients and mice with genetic mutation for the disorder Usher syndrome, researchers from the University of Maryland School of Medicine (UMSOM), the National Institutes of Health’s National Eye Institute (NEI), and National Institute on Deafness and Other Communication Disorders (NIDCD), documented the natural history of vision...
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...
Rockville, MD, USA, and Pratteln, Switzerland – ReveraGen Biopharma and Santhera Pharmaceuticals (SIX: SANN) announce that ReveraGen has received a USD 1.2 million grant from the FDA under their “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)” grants program. The grant adds to existing grants from...
San Diego, California – In recent years, microbiome research has started to shift its focus from the microbes themselves to the molecules they produce. After all, it’s these molecules that directly interact with human cells to influence a person’s health. But trying to identify which molecules are being made by...
Beijing, China – Patients with lupus nephritis (LN), a severe complication of systemic lupus erythematosus (SLE), often undergo progressive kidney damage, with approximately 20% of these patients advancing to end-stage renal disease. The current therapeutic landscape for LN, dominated by glucocorticoids and immunosuppressants, is limited by suboptimal response rates, the...