Skip to main content
Skip to main menu
UF Health
University of Florida
Powell Center
for
Rare Disease Research and Therapy
Department
of
Pediatrics
in
the
College
of
Medicine
Support our Mission
Open Search
Open Main Menu
Main Navigation
Home
About Us
Show submenu for 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
Clinical Research Center
Show submenu for 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
Show submenu for 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
Navigating Gainesville
Show submenu for Navigating Gainesville
Navigating Gainesville
Overview
Air Travel
Accessible Transport Rentals
Overnight Accommodations
Dining Experience
Fun Around Town
Fun Around Florida
News
Contact Us
All Posts
Neuromuscular Diseases
Posted in
Neuromuscular Diseases
Roche/Genentech Announces Decision to Discontinue Development of RG6206 (RO7239361)
November 6, 2019
Solid Biosciences Reports First Quarter Financial Results and Study Update
May 21, 2019
2019 PPMD Conference Agenda
May 14, 2019
PPMD Duchenne Registry: Researchers Analyze Steroid Use
May 9, 2019
New Duchenne Muscular Dystrophy Guide for Families
September 13, 2018
FDA Announces the Approval of TIGLUTIK in ALS
September 11, 2018
MDA Announces 34 New Research Grants Totaling $9.9 million
September 10, 2018
Medicine at the mall: UF Health opening specialty practices, surgical center
June 7, 2018
UF students raise more than $3 million for Dance Marathon
March 26, 2018
AMO Pharma’s Myotonic Dystrophy Therapy Improves Cognition and Function
March 21, 2018
First
Prev
1
2
3
4
5
Next
Last