Troy, New York – Using artificial intelligence tools to analyze years of biomedical data, researchers at Rensselaer Polytechnic Institute have discovered a possible connection between sleep, gastrointestinal health, and two potentially harmful behaviors often associated with profound autism: self-injury and aggression. Their study is published in the Journal of Personalized...
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Leipzig, Germany – Every year, around 4,000 people in Germany are diagnosed with myelodysplastic neoplasms (MDS). The disease can lead to anaemia, infections and an increased risk of bleeding. People with higher-risk MDS have a significantly lower life expectancy than the average person of the same age. In the higher-risk...
MIAMI, Florida — Resolve Therapeutics, LLC, a mid-stage clinical development biotechnology company pioneering first-in-class therapies for autoimmune diseases, today announced the publication of results from its phase 2a clinical trial of RSLV-132 in patients with Systemic Lupus Erythematosus (SLE) in Lupus Science & Medicine. The publication entitled “Evaluation of RNase...
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...
MILAN, Italy – Beta-thalassemia (Bthal) is a genetic disorder due to mutations in the ß-globin gene, leading to a reduced or absent production of HbA, which interferes with erythroid cell maturation and limits red cell production. Patients are affected by severe anemia, hepatosplenomegaly, and skeletal abnormalities due to rapid expansion...
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...