American Council on Science and Health3d
“Rare” but Not Uncommon: The Hidden Crisis of Rare Diseases
Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...
Psychology Today7d
From Anecdotes to Data: Researching Sleep in a Rare Disorder
Rare diseases are sometimes called “orphan diseases,” referring to the fact that they are frequently understudied in the ...
CURE3h
FDA Grants Orphan Drug Designation to PEP-010 in Pancreatic Cancer
The FDA has granted orphan drug designation to PEP-010 for pancreatic cancer, advancing its development as a potential novel ...
Opinion
BusinessWorld8dOpinion
Transforming rare disease outcomes
Reivi Dela Cruz was only four years old when he was diagnosed with Neurofibromatosis Type 1 (NF1), a genetic condition that causes tumors (neurofibromas, which are usually non-cancerous) to grow on or ...
The Herald-Zimbabwe13d
Spotlight on rare diseases
Even when a diagnosis is made, treatment remains elusive. Medications for rare diseases—known as orphan drugs—are prohibitively expensive and rarely available in Zimbabwe, forcing families to ...
BioArctic receives Orphan Drug Designation for exidavnemab the US
BioArctic AB (publ) (Nasdaq Stockholm: BIOA B) announced today that the US FDA Office of Orphan Products Development (OOPD) has granted orphan drug ...
PTC Therapeutics: Upside In Rare Disease Innovation
PTC Therapeutics could boost revenue with vatiquinone for Friedreich's ataxia. Find out why PTCT stock is poised for growth ...
Morningstar13d
The US FDA Grants Both Orphan Drug and Rare Pediatric Disease Designations to NEU-001 of Neurenati for the Treatment of Hirschsprung Disease
The US FDA Grants Both Orphan Drug and Rare Pediatric Disease Designations to NEU-001 of Neurenati for the Treatment of Hirschsprung Disease Neurenati Therapeutics Inc., a biotech company focused ...
Mena FN7d
11.8% CAGR Orphan Drugs Market Is Projected To Gain $4,35,686.3 Million By 2030
With advancements in biotechnology and personalized medicine, orphan drugs offer hope to patients and families affected by rare diseases, improving their quality of life and life expectancy.
Quoin Pharmaceuticals, Inc.: Quoin Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full-Year 2024 Financial Results
Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a late clinical-stage specialty pharmaceutical company focused on rare and orphan ...
Yahoo Finance13d
The US FDA Grants Both Orphan Drug and Rare Pediatric Disease Designations to NEU-001 of Neurenati for the Treatment of Hirschsprung Disease
has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to NEU-001, a novel combination therapy utilizing a neurotrophic growth factor, for the treatment of ...