Wayne, Pa. — Palvella Therapeutics, Inc., a late clinical-stage biopharmaceutical company whose vision is to become the leading rare disease company focused on developing and commercializing novel therapies to treat individuals suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies, announced positive topline results from...
treatment News
MINNEAPOLIS — Panbela Therapeutics, Inc., a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs announces the publication of clinical data from studies of CPP-1X (also known as α-Difluoromethylornithine (DFMO) or Eflornithine) in neuroblastoma. According to Hogarty et al, children with relapsed...
Houston, TX – The use of high-protease pancreatic replacement therapy demonstrated improvement in maladaptive behaviors, such as irritability, in preschool children with autism spectrum disorder (ASD), according to research conducted at 32 clinical sites, including UTHealth Houston. The study was published recently in JAMA Network Open. “Children who have ASD...
PITTSBURGH, PA — PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce it was selected by Chiesi Global Rare Diseases, a business unit of the Chiesi Group, for the distribution of Filsuvez® topical gel, the first approved treatment for...
CHICAGO – Emalex Biosciences, Inc. (“Emalex”), a biopharmaceutical company founded by Paragon Biosciences to develop treatments for central nervous system movement disorders and fluency disorders, today announced positive topline results from its Phase 2b clinical study (D1AMOND Study) evaluating the efficacy and safety of ecopipam (EBS-101), an investigational, first-in-class, dopamine-1 (D1) receptor...
CAMBRIDGE, Mass. – Paragonix Technologies, Inc. is announcing the release of the recently developed Paragonix Web Portal, an accessible support platform that connects with the Paragonix App and donor organ preservation devices to provide transplant teams the ability to digitally participate in live sessions or review historical cases and clinical...
MONTEREY, Calif. — This month’s Journal of Aerosol Medicine and Pulmonary Drug Delivery features positive Phase Ib clinical trial results for PARI Pharma’s L-CsA, inhaled liposomal cyclosporine A. In May, PARI Pharma received Orphan Drug Designation from the Food and Drug Administration for L-CsA, delivered via an Investigational eFlow Nebulizer...
SOUTH SAN FRANCISCO, Calif. and MIAMI, Florida — Pasithea Therapeutics Corp. (NASDAQ: KTTA), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) and other cancer indications, today announced the activation of four clinical trial sites in the United States. These...
Hong Kong – Amyotrophic Lateral Sclerosis (ALS) is a fatal type of motor neuron disease characterized by progressive degeneration of nerve cells in the spinal cord and brain, resulting in muscle weakness and paralysis that can include the inability to walk and speak, or even swallow and breathe. The average...
MIAMI, FLORIDA – In a study led by researchers at Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine, patients recently diagnosed with a common and aggressive form of acute myeloid leukemia reported having improved quality of life when a newly approved drug was part of...