Sarepta Therapeutics Announces Intent to Submit an Accelerated Approval Biologics License Application for its Gene Therapy SRP-9001 to Treat Duchenne Muscular Dystrophy
CAMBRIDGE, Mass., July 29, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced its intent to submit a Biologics License Application (BLA) seeking accelerated... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - July 29, 2022 Category: Pharmaceuticals Source Type: clinical trials
Soligenix Receives FDA IND Clearance for Phase 2 Clinical Trial of Synthetic Hypericin in the Treatment of Psoriasis
PRINCETON, N.J., June 28, 2022. Soligenix, Inc. (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - June 28, 2022 Category: Pharmaceuticals Source Type: clinical trials
Clincierge Releases Study on Patient Experience in Clinical Trials
PHILADELPHIA and SAN DIEGO , June 14, 2022. Clincierge today released " Voice of the Patient, " a research report examining the patient experience in clinical trials of rare diseases, at the 2022 BIO International Convention in San Diego,... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - June 14, 2022 Category: Pharmaceuticals Source Type: clinical trials
SpringWorks Therapeutics Announces Nirogacestat Achieved Primary and All Key Secondary Endpoints in Phase 3 DeFi Trial in Adult Patients with Progressing Desmoid Tumors
STAMFORD, Conn., May 24, 2022 (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 - May 24, 2022 Category: Pharmaceuticals Source Type: clinical trials
Verification of the Epidemiology and Mortality of Rare Diseases in Taiwan With Real-world Evidence
Conditions: Rare Diseases; Epidemiology; Morbidity; Morality; Comorbidities and Coexisting Conditions; Health Care Utilization Intervention: Other: longitudinal observational study Sponsor: National Cheng-Kung University Hospital Completed (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - May 10, 2022 Category: Research Source Type: clinical trials
