Patient Assistance & Treatment Resources

Note: Information on Thalassemia and Sickle Cell Anemia may be included because these diseases are similar hemolytic anemias.

Patient Assistance

National Institute of Health Clinical Trials Database Search / ClinicalTrials.gov

Free genetic testing through Anemia ID

myAgios Patient Support Services

Novartis Jadenu (medication) Assistance Program

GoodRx Prescription Drug Savings Card

Financial Aid for Patients & Families (NIH)

Medicaid & CHIP Health Insurance

Social Security Disability Income Application

Social Security Disability Evaluation for Hematological Disorders in Adults

Social Security Disability Evaluation for Hematological Disorders in Children

Treatment Resources

Lentiviral gene therapy for pyruvate kinase deficiency (video) (2021)

Diagnosis, monitoring, and management of pyruvate kinase deficiency in children (2022)

Genotype-Response Correlation In Drive PK, A Phase 2 Study Of Mitapivat (AG-348) In Patients With Pyruvate Kinase Deficiency (2019)

Splenectomy and emerging novel treatments in rare inherited hemolytic anemias (2019)

Report an Adverse Event or Serious Problem to the FDA

Study Opportunities

Gene Therapy (Pediatric) / start date was July 6, 2020

Mitapivat in Pediatric Patients / start date will be March 1, 2022

Mental Healthcare

Find a Therapist / Psychology Today

10 Young People Changing Their Communities For Mental Health / Mental Health America


Are we missing anything?

If you have a great PKD resource to add to our collection, please contact us at twpkdnonprofit@gmail.com with the title and a public link!