American Council on Science and Health2d
“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 ...
Opinion
BusinessWorld7dOpinion
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 Manila Times3d
Quoin Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full-Year 2024 Financial Results
Investors are encouraged to read the Company's Annual Report on Form 10-K when it is filed with the Securities and Exchange ...
Labiotech.eu13d
Rare Disease Day: seven drugs awaiting approval in 2025
As we observe Rare Disease Day today, let us take a look at seven drugs poised to be approved to address rare diseases in ...
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 ...
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 ...
Yahoo Finance12d
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 ...
Mena FN6d
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.
The Manila Times12d
Quoin Pharmaceuticals Files U.S. and International Patent Applications for Novel Topical Rapamycin Formulations to Treat Rare Disease Indications
Proprietary Invisicare Delivery Technology Designed to Optimize Local Skin Penetration The proprietary Invisicare delivery technology, which Quoin has exclusive rights to for all orphan rare skin ...
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 ...