Patient Assistance & Treatment Resources

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

Patient Assistance

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

Center for Parent Information & Resources on Education for Children with Disabilities


Nutrition for Patients with Thalassemia & Pyruvate Kinase Deficiency (2023)

Gene & Cell Therapy

Rocket Pharma Gene Therapy Powerpoint Information (2022)

Gene & Cell Therapy Education

Lentiviral gene therapy for Pyruvate Kinase Deficiency (video) (2021)

Study Opportunities

Clinical Trials

Peak Registry Study /in progress

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

Mitapivat in Pediatric Patients / 2022

Mental Healthcare

Tools for Student Mental Health (2022)

Addiction & Mental Health Resources:  Live Another Day

Find a Therapist / Psychology Today

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

Report an Adverse Event or Serious Problem to the FDA

Are we missing anything?

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