Cerebral Creatine Deficiency Syndromes (CCDS) EL-PFDD Meeting скачать в хорошем качестве

Cerebral Creatine Deficiency Syndromes (CCDS) EL-PFDD Meeting

The Association for Creatine Deficiencies is holding an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting on Cerebral Creatine Deficiency Syndromes (CCDS). This virtual meeting is an important opportunity for patients and caregivers to inform FDA representatives, academic and scientific researchers, medical professionals, and pharmaceutical companies about personal experiences regarding the symptoms and daily impact of CCDS, as well as thoughts on current and future approaches to therapies. The PFDD Program was created by the FDA several years ago as a way to systematically gather information from patients and caregivers about their conditions, especially symptoms and daily impact, as well as thoughts on current and future approaches to therapies. This information helps inform FDA’s drug development decision making process. The Association for Creatine Deficiencies is thankful for FDA’s approval of our proposal, recognizing the importance of learning about the three rare genetic disorders that fall under CCDS. Externally-Led PFDD’s are hosted by an organization other than the FDA, in this case the ACD.