În prezent, recrutează studii clinice / studii pentru acidemia propionică
Istorie naturală NIH, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
This study will evaluate patients with propionic acidemia to learn more about the genetic and biochemical causes and the medical complications associated with it. De asemenea, planul de a studia modul in care bacteriile care trăiesc în intestin nostru (microbiome) can affect the course of propionic acidemia.
Studiu Open Label al ARNm-3927 la pacienții cu acidemie propionică
This is a Phase 1/2 study sponsored by Moderna TX, Inc. It is designed to evaluate if an investigational treatment called mRNA-3927 is safe and effective in reducing the symptoms of PA in individuals one year of age and older. mRNA 3927 este un intravenos investigativ (IV) tratament perfuzabil care instruiește corpul unei persoane să producă o enzimă PCC care funcționează.
De la 4/23/23, the study is recruiting at the following sites:
Statele Unite:
- David Geffen School of Medicine UCLA
- Johns Hopkins Hospital
- Boston Children's Hospital
- University of Michigan
- Școala de Medicină Icahn de pe Muntele Sinai – Clinical Research Unit
- Duke University Medical Center
- Cincinnati Children’s Hospital
- Spitalul de Copii din Philadelphia
- Texas Children’s Hospital
Canada:
- Hospital For Sick Children
United Kingdom:
- University Hospital Birmingham NHS Foundation Trust
- Great Ormond Street Hospital (GOSH)
Review of Charts From Amish/Mennonite Variant PA Patients
The study is designed to provide a comprehensive description of the clinical and biochemical features of propionic acidemia, Amish/Mennonite variant (PA-AMV).
Recruiting at Children’s Hospital of Pittsburgh
DISCLAIMER
Propionic Acidemia Foundation website is designed for educational purposes only and is not intended to serve as medical advice. Informațiile furnizate pe acest site nu ar trebui să fie utilizate pentru diagnosticarea sau tratarea unei probleme de sanatate sau de boala. Acesta nu este un substitut pentru îngrijire profesională. If you suspect that you or your children may have Propionic Acidemia, you should consult your health care provider. Any potential therapy should be thoroughly discussed with your medical provider. The Propionic Acidemia Foundation does not recommend nor endorse any particular products, terapeutică, companies, or manufacturers.
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