Skip to main content Skip to main menu
UF Health UF Health Monogram University of Florida
UF monogram
Powell Center for Rare Disease Research and Therapy Department of Pediatrics in the College of Medicine
Support our Mission

Main Navigation

  • Home
  • About Us - current page
    • About Us Overview
    • Meet the Director
    • Publications
    • Gallery
    • Helpful Links
      • Clinical Research Sponsors
      • Patient Organizations and Foundations - current page
      • Additional Caregiver Resources
      • Rare Disease Podcasts
  • Clinical Research Center
    • Clinical Research Center Overview
    • Research Team
    • Research Space
    • Clinical Trials
      • Amyotrophic Lateral Sclerosis
      • Barth Syndrome
      • Duchenne Muscular Dystrophy
      • Friedreich's Ataxia
      • Mucopolysaccharidosis Type II
      • Myotubular Myopathy
      • Neuromuscular Diseases
      • Pompe Disease
  • Neuromuscular Clinic
    • Neuromuscular Clinic Overview
    • Clinical Team
    • Care Center Space
    • Conditions We Treat
    • Commercial Treatments
      • Emflaza (Deflazacort)
      • Evrysdi (risdiplam)
      • Lumizyme (alglucosidase alfa)
      • Spinraza (Nusinersen)
      • Zolgensma (Onasemnogene abeparvovec)
      • VYONDYS 53 (Golodirsen)
      • EXONDYS 51 (Eteplirsen)
    • Diagnostic Testing
    • Patients & Families
      • Preparing For Your Appointment
      • MyUFHealth (MyChart)
      • Genetic Testing
      • Applying for Disability
      • COVID-19
      • Influenza (Flu) Season
    • Referrals
    • Research
  • Navigating Gainesville
    • Navigating Gainesville Overview
    • Air Travel
    • Accessible Transport Rentals
    • Overnight Accommodations
    • Dining Experience
    • Fun Around Town
    • Fun Around Florida
  • News
  • Contact Us
  1. About Us
  2. Helpful Links
  3. Patient Organizations and Foundations
About Us Overview
  • Meet the Director
  • Publications
  • Gallery
  • Helpful Links
    • Clinical Research Sponsors
    • Patient Organizations and Foundations
    • Additional Caregiver Resources
    • Rare Disease Podcasts

Patient Organizations and Foundations

Jett Foundation

These groups—many founded by parents within the rare disease community—help connect patients and families to education, research, and support services. Here are just a few of the organizations that work to raise both awareness of rare diseases and funds directed toward research while supporting their communities.

Muscular Dystrophy Association

LEARN MORE
MDA Logo

Parent Project Muscular Dystrophy

LEARN MORE
PPMD

Cure Duchenne

LEARN MORE
Cure Duchenne

Jett Foundation

LEARN MORE
Jett Foundation

Little Hercules Foundation

LEARN MORE
Little Hercules

Team Joseph

LEARN MORE
Team Joseph

Charley’s Fund

LEARN MORE
Charley's Fund

Hope for Gabe

LEARN MORE
H4G

Joining Jack

LEARN MORE
Joining Jack

Duchenne UK

LEARN MORE
Duchenne UK

Cure Spinal Muscular Atrophy

LEARN MORE
Cure SMA

Friedreich’s Ataxia Research Alliance

LEARN MORE
FARA

MTM CNM Family Connection

LEARN MORE
MTM CNM

Joshua Frase Foundation

LEARN MORE
Joshua Frase Foundation

Barth Syndrome Foundation

LEARN MORE
Barth Syndrome

Canavan Foundation

LEARN MORE
Canavan Foundation

Chasing Connor’s Cure

LEARN MORE
Chasing Connor's Cure

Asociación Española de Enfermos de Pompe

LEARN MORE
AEE Pompe

Cure Congenital Muscular Dystrophy

LEARN MORE
Cure CMD

Charcot-Marie-Tooth Association

LEARN MORE
CMT

National Ataxia Foundation

LEARN MORE
National Ataxia Foundation

United Mitochondrial Disease Foundation (UMDF)

LEARN MORE
United Mitochondrial

Myasthenia Gravis Foundation of America

LEARN MORE
Myasthenia Gravis Foundation

RYR-1 Foundation

LEARN MORE
RYR-1 Foundation

UF monogram
Powell Center for Rare Disease Research and Therapy Department of Pediatrics in the College of Medicine

Utility Links

  • Pediatrics Department
  • Powell Gene Therapy Center
Physical Address
Clinical and Translational Research Building
2004 Mowry Road, Gainesville, FL 32610
Gift Icon

Make a Gift

Giving Opportunities
Social Media
Facebook Twitter
University of Florida Health - Home UF Health Logo
University of Florida - Home UF Logo
  • Log in
  • Website Support
  • Sitemap
  • Disclaimer & Permitted Use
  • Disability Services
  • Security Policies
  • UF Privacy Policy
  • Shands Privacy Policy
  • This page uses Google Analytics (Google Privacy Policy)

© 2025 University of Florida Health