Currently Recruiting Clinical Trials/Studies For Propionic Acidemia

Natural History, 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.  They also plan to study how bacteria living in our gut (microbiome) can affect the course of propionic acidemia.

Hemoshear Insight Study – HemoShear is now recruiting participants for the MMA and PA Insight Study. The study will evaluate how different assessments can measure verbal communication, processing speed, life skills, quality of life and sleep. The study is conducted virtually – no clinic visits are required.

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia (HERO)

Tuesday, April 13, 2021 1:00 pm – 2:00 pm EST – Introducing HST5040 Being Developed for Methylmalonic and Propionic Acidemias (for clinicians) – presented by HemoShear Therapeutics

Saturday, April 17, 2021 1:00 pm CDT – PAF/ OAA  invite you to  Caregiver Webinar on New Horizons for MMA/PA: Current Treatments, Clnical Trials and New Approaches –

Speakers:
Nicola Longo, MD.,  University of Utah School of Medicine
Kimberly Chapman, MD, PhD, Children’s National Hospital, Washington, D.C.
Gerry Cox, MD, PhD, HemoShear Therapeutics, Chief Medical Officer
Brian Wamhoff, PhD, HemoShear Therapeutics, Co-Founder and Chief Operating Officer

 

Open Label Study of mRNA-3927 in Patients With Propionic Acidemia

The Paramount Study is a Phase 1/2 study.  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 is an investigational intravenous (IV) infusion treatment that instructs a persons’ body to make a PCC enzyme that works.

Saturday, April 10, 2021  11:00 am – 12:30 EST  – PAF/OAA invite you to Advancing mRNA to Treat Organic Acidemias – Informational Webinar on Moderna’s upcoming treatment and clinical trial.

Speakers:
Matthew Lumley, Moderna, Senior Director, Rare Disease Clinical Development
Stephen Gleason, Moderna, Director, Clinical Operations

Registration Link

DISCLAIMER

Propionic Acidemia Foundation website is designed for educational purposes only and is not intended to serve as medical advice.  The information provided on this site should not be used for diagnosing or treating a health problem or disease.  It is not a substitute for professional care.  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, therapeutics, companies, or manufacturers.