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 ...
Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...
Anecdotal evidence has long suggested that sleep problems are especially common for people with Moebius syndrome. Now, a new ...
PTC Therapeutics could boost revenue with vatiquinone for Friedreich's ataxia. Find out why PTCT stock is poised for growth ...
Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström ...
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 European orphan drug market is growing and is already comparable in size and scope to the US market. The European Union ...
Post-authorisation safety studies (PASS) are often seen simply as a regulatory necessity, a checkbox that ensures ongoing ...
High-level results from the CALYPSO Phase III trial showed that eneboparatide (AZP-3601), an investigational parathyroid hormone (PTH) receptor 1 agonist, met its primary endpoint with statistical ...
READ: Rare, deadly diseases in PH get attention from barely enforced law According to the Philippine Society for Orphan Disorders (PSOD), the lack of widespread awareness, limited funding ...
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 ...
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