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...
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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,...
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...
Alexandria, VA. Patients in the phase 3 CheckMate-649 trial (NCT02872116) who received nivolumab (Opdivo) plus chemotherapy reported stable or better on-treatment health-related quality of life (HRQOL) compared with chemotherapy alone. CheckMate-649 included patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, or esophageal adenocarcinoma, and findings were published in...