FDA Issues Ethical Guidance to Protect Children who Participate in Clinical Trials
Rare Daily Staff The U.S. Food and Drug Administration issued a draft guidance to assist industry, sponsors and institutional review
FDA Places Clinical Hold on Pharvaris HAE Clinical Trials in the United States
Rare Daily Staff Pharvaris said the U.S. Food and Drug Administration verbally informed the company that, based on its review
ProQR Winds Down Clinical Trials to Focus Exclusively on Axiomer RNA-editing Technology
Rare Daily Staff ProQR Therapeutics, following the European Medicines Agency recommendation that the company conduct an additional phase 3 study
Designing Clinical Trials with the Patient in Mind
Jenn McNary is a mother of children with rare conditions, as well as an outspoken advocate who has sought to
Keeping Clinical Trials Running Smoothly
Clinical trials can get derailed for a variety of reasons that may have nothing to do with whether a drug
RARE Drug Development Symposium: Disruptive Innovations in Clinical Trials Workshop
The 2022 RARE Drug Development Symposium kicks off June 8, and Global Genes is ecstatic about the speaker lineup for
Getting Your Community Ready for Clinical Trials
Helping patients and caregivers understand their options for clinical trials – and what to consider when making a decision on
BioCryst Pauses Enrollment in BCX9930 Clinical Trials
Rare Daily Staff In a two-sentence statement, BioCryst Pharmaceuticals said it had paused enrollment in three clinical trials of its
FDA Awards 11 Grants to Clinical Trials to Develop New Medical Products for Rare Disease Treatments
Rare Daily Staff The U.S. Food and Drug Administration has awarded 11 new clinical trial research grants totaling more than
IndoUSrare Webinar “Clinical Trials: The Hope to Cure Rare Diseases” by Mohamed Duklef
We are incredibly excited to present to you, “Clinical Trials: The Hope to Cure Rare Diseases” by Mohamed Duklef. The
Stealth Bio Raises $30 Million to Advance Elamipretide Clinical Trials
Rare Daily Staff Stealth BioTherapeutics, a clinical-stage biotechnology company focused discovering, developing, and commercializing novel therapies for diseases involving mitochondrial
Putting the Patient at the Center of Rare Disease Clinical Trials
The recruitment of patients for rare disease clinical trials can be challenging because of issues such as small patient populations
