US Food and Drug Administration has cleared HemoShear to proceed with a clinical trial of their investigational drug HST5040 for the treatment of MMA and PA.
- Letter to MMA and PA Community
- HemoShear Receives IND clearance for HST5040 for metabolic diseases June…
PAF awards $44,253 New Grant in 2020
Rajavel Elango, PhD, University of British Columbia
“Optimizing amino acids in medical foods to manage propionic acidemia”
PAF Awards $30,591 Continuation Grant in 2020
Eva Richard, PhD, Universidad Autonoma de Madrid, Spain
“Cardiomyocytes derived from induced pluripotent stem cells as a new model for therapy development in propionic acidemia”
2020 PA FAMILY DAY (postponed due to Covid-19) – hoping to reschedule
Spring newsletter is available.
Fall stories/articles due August 15th, 2020. Please email to [email protected] Please register with PAF if you would like to receive the newsletter by mail or email or update your contact information.
Due October 1, 2020 – For questions, contact [email protected]
PROP Nutrition Guidelines and Toolkit are now available. NMG PROP announcement Final
PROP Nutrition Guidelines and Toolkit are now available online. The Nutrition Management Guideline for individuals with propionic acidemia (PROP) is part of a larger project undertaken by the Southeast Regional Newborn Screening and Genetics Collaborative (SERC) (HRSA Region 3) and Genetic Metabolic Dietitians International (GMDI) to develop nutrition management guidelines for inherited metabolic disorders (IMD).
PA International Patient Registry
The PA International Patient Registry provides a way for PA patients and families worldwide to improve the understanding of the disorders and accelerate research by reporting information about how the disorder affects them. The Registry is itself an IRB-approved research project and the data collected will help characterize the condition of people living with PA. You can find out more on www.paregistry.org
Pounding the Pavement for PA
Join the PAF Pounding the Pavement for PA team or start your own! Run in any race, around your block, or become a “virtual runner” Contact Brittany Smith for more information at [email protected] . Check out events to follow our progress.