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
    • About Us Overview
    • Meet the Director
    • Publications
    • Gallery
    • Helpful Links
      • Clinical Research Sponsors
      • Patient Organizations and Foundations
      • Additional Caregiver Resources
      • Rare Disease Podcasts
  • Navigating Gainesville
    • Navigating Gainesville Overview
    • Air Travel
    • Accessible Transport Rentals
    • Overnight Accommodations
    • Dining Experience
    • Fun Around Town
    • Fun Around Florida
  • Clinical Research Center
    • Clinical Research Center Overview
    • Research Team
    • Research Space
    • Clinical Trials
      • Amyotrophic Lateral Sclerosis
      • Barth Syndrome
      • Becker Muscular Dystrophy
      • Duchenne Muscular Dystrophy
      • Friedreich's Ataxia
      • Mucopolysaccharidosis I
      • 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)
      • EXONDYS 51 (Eteplirsen)
      • VYONDYS 53 (Golodirsen)
      • Zolgensma (Onasemnogene abeparvovec)
    • Diagnostic Testing
    • Patients & Families
      • Preparing For Your Appointment
      • MyUFHealth (MyChart)
      • Genetic Testing
      • Applying for Disability
      • COVID-19
      • Influenza (Flu) Season
    • Referrals
    • Research
  • News
  • Contact Us
  1. All Posts
  2. Archive

All Posts

Smith receives James Dunleavy Distinguished Service Award

February 16, 2021

People

Santhera Shares Community Update on Phase 2b Trial for Vamorolone

February 1, 2021

Scientist

Provention Bio Announces Key Findings from Pre-clinical Proof-of-concept Study

January 28, 2021

Prevention Bio

Sarepta Therapeutics Announces Results for Part 1 of Study Evaluating SRP-9001

January 7, 2021

DNA

Pfizer doses the first participant in the Phase 3 CIFFREO study

January 7, 2021

Gene Therapy

REGENXBIO Announces New Gene Therapy Program for Duchenne

January 5, 2021

Regenx

Sarepta Therapeutics Announces Positive Clinical Results from MOMENTUM

December 7, 2020

Sarepta

Denison Magazine: The Idea Guy

November 2, 2020

Barry Byrne

Antisense Therapeutics Receives FDA Orphan Drug Designation for ATL1102

November 2, 2020

Disabled boy in wheelchair

Antisense Therapeutics Receives US FDA Orphan Drug Designation for ATL1102

October 26, 2020

ATL

Recent News

  • Smith receives James Dunleavy Distinguished Service Award February 16, 2021
  • Santhera Shares Community Update on Phase 2b Trial for Vamorolone February 1, 2021
  • Provention Bio Announces Key Findings from Pre-clinical Proof-of-concept Study January 28, 2021
First Prev
1 2 3 4 5
Next Last
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)

UF Health is a collaboration of the University of Florida Health Science Center, Shands hospitals and other health care entities.

© 2021 University of Florida Health