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Peking Union Medical College Hospital — NA
University of Oxford
Aga Khan University — NA
University of Rochester
Hacettepe University
Larry W. Markham — PHASE1
Lokman Hekim University — NA
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
Total programs
Open now
Foundation grants
Copay cards
Travel grants
Foundation Grants7
NORD Patient Assistance — Duchenne muscular dystrophy
NORD Patient Assistance
Good Days — Duchenne muscular dystrophy
Good Days
HealthWell Foundation — Duchenne muscular dystrophy
HealthWell Foundation
The Assistance Fund — Duchenne muscular dystrophy
The Assistance Fund
Patient Services Inc — Duchenne muscular dystrophy
Patient Services Inc
Patient Advocate Foundation — Duchenne muscular dystrophy
Patient Advocate Foundation
PAN Foundation — Duchenne muscular dystrophy
PAN Foundation
Copay Assistance6
Patient Assistance Programs3
Travel Grants16
Assistance Fund: Duchenne Muscular Dystrophy
The Assistance Fund
Cranbury Connects Patient Assistance Programs
Duchenne Muscular Dystrophy Copay Assistance Grant
Duchenne Muscular Dystrophy Disease Fund
PAN Foundation
Duchenne Muscular Dystrophy Financial Assistance Program
The Assistance Fund
Emflaza (deflazacort) Copay Assistance
PAN Foundation Duchenne Muscular Dystrophy Copay Assistance
PAN Foundation
PAN Foundation Duchenne Muscular Dystrophy Copay Assistance Fund
PAN Foundation
PAN Foundation Duchenne Muscular Dystrophy Copay Assistance Grant
PAN Foundation
PAN Foundation Duchenne Muscular Dystrophy Disease Fund
PAN Foundation
PAN Foundation Duchenne muscular dystrophy fund
PAN Foundation
QuickRx Emflaza (deflazacort) Copay Assistance
SareptAssist
The Assistance Fund (TAF) Duchenne Muscular Dystrophy Financial Assistance Program
The Assistance Fund
The Assistance Fund Duchenne Muscular Dystrophy Financial Assistance Program
The Assistance Fund: Duchenne Muscular Dystrophy
Deflazacort Oral Suspension
(DEFLAZACORT)Orphan drugZydus Pharmaceuticals (USA) Inc.
ELEVIDYS
(DELANDISTROGENE MOXEPARVOVEC-ROKL)Orphan drugSarepta Therapeutics, Inc.
Vyondys 53
(GOLODIRSEN)Orphan drugSarepta Therapeutics, Inc.
AMONDYS 45
(CASIMERSEN)Orphan drugSarepta Therapeutics, Inc.
Viltepso
(VILTOLARSEN)Orphan drugNS Pharma, Inc.
Deflazacort Oral Suspension
(DEFLAZACORT ORAL)Orphan drugSun Pharmaceutical Industries, Inc.
DUVYZAT
(GIVINOSTAT)Orphan drugItalfarmaco SPA
AGAMREE
(VAMOROLONE)Orphan drugCatalyst Pharmaceuticals, Inc.
The post-pandemic pivot: mRNA therapeutics enter the chronic rare disease arena
Published in Mol Ther Nucleic Acids. Giangrande PH et al.
CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disorder caused by loss of dystrophin. Upregulating utrophin, a dystrophin paralog, is a promising gene therapy approach. Here, we present a...
Treatment advances for Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a severe X-linked muscle wasting disease with childhood onset that leads to loss of ambulation in the teenage years and mortality in the second or third decade of ...
Serum biomarkers in Duchenne muscular dystrophy: a leap toward precision diagnostics.
I was interested to read the article by Dowling et al.1 in the European Journal of Translational Myology, where the authors provide a comprehensive review of serum biomarkers for Duchenne Muscular Dys...
Adeno-Associated Virus Toxicity in Duchenne Muscular Dystrophy: Mechanisms and Clinical Considerations.
Background/Objectives : Recombinant adeno-associated virus (AAV) vectors have revolutionized gene therapy for monogenic diseases such as Duchenne muscular dystrophy (DMD). However, high systemic doses...
The impact of antisense oligonucleotide (ASO) therapeutics on the future of rare disease drug discovery
Published in Expert Opin Drug Discov. Ruchi R et al.
Francesco Muntoni, Professor
UCL GOS Institute of Child Health
📍 London
Jiwen Wang
Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
📍 Shanghai, Shanghai Municipality
Marika Pane
IRCCS Eugenio Medea - Ass. "La Nostra Famiglia"
📍 Bosisio Parini, Lc
JungHyun Kim, prof
Seoul National University Hospital
📍 Seoul, Jongno
Craig McDonald, MD
Phoenix Children's Hospital
📍 Phoenix, Arizona
Tanja Taivassalo
University of Florida
📍 Gainesville, Florida