Moderna Announces First Patient Dosed in Phase 1/2 Study of mRNA-3705 for Methylmalonic Acidemia
mRNA-3705 is Moderna’s second rare disease program to enter clinical studies
mRNA-3705 granted Orphan Drug and Rare Pediatric Disease designation by the U.S. FDA
MMA associated with significant mortality and morbidity; there are no... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - August 16, 2021 Category: Pharmaceuticals Source Type: clinical trials
SpringWorks Therapeutics Announces Phase 1b/2a Clinical Trial of Mirdametinib in Patients with Advanced Solid Cancers Harboring MAPK-Activating Mutations
STAMFORD, Conn., Aug. 03, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - August 3, 2021 Category: Pharmaceuticals Source Type: clinical trials
Protalix BioTherapeutics and Chiesi Global Rare Diseases Receive Complete Response Letter for Pegunigalsidase Alfa from FDA
CARMIEL, Israel and BOSTON, April 28, 2021 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - April 28, 2021 Category: Pharmaceuticals Source Type: clinical trials
FACE for Children With Rare Diseases
Condition: Rare Diseases Intervention: Behavioral: FAmily CEntered (FACE) pACP Intervention Sponsors: Children's National Research Institute; Respecting Choices; National Institutes of Health (NIH); National Institute of Nursing Research (NINR) Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - April 22, 2021 Category: Research Source Type: clinical trials
