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...
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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...
MIDDLETOWN, Ohio — Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced that PPMD will be honoring United States Senator Johnny Isakson and the American Football Coaches...
Staying alive continues to be a day-to-day struggle for little Billy Russell as his health deteriorates due to the rare disease, Charge syndrome. The Daily in April revealed the plight of Lisa and Dean Russell, the parents of eight-month-old Billy, who were struggling to make ends meet and care for...
When little Callum Miles was born with a rare genetic disorder, his parents were told he would probably have to go to a special needs school. But two years after his mum and dad fought to get him into nursery, the four-year-old is now looking forward to starting at a...
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...
Parker Pennington won’t be at the June 27 Skate for Life exhibition showcasing his jumping skills or performing the routine that had him sitting in third place after the short program at the U.S. Figure Skating Championships in Cleveland earlier this year. He will, however, host the charity event for...
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For this reason,...