This is a growing compilation of drugs that are in the pipeline for Duchenne Muscular Dystrophy medication or cure. being an ongoing, energetic project this number will gain out of your feedback, so please enable me know of any updates or suggestions. being a substance moves by way of the pipeline, i will update the status and provide only the most recent trial information-this will simplify the number to only the most pertinent information. For example, Ataluren is only mentioned in the phase III section since it has made it by way of the previous trials which are completed, suspended, or terminated for various reasons. The complete medical historical past of the following drugs can be searched at ClinicalTrials.gov. A well-organized compilation of research work also exists on the cure Duchenne research Index page. These work are intended to understand the on the market selections and the magnitude of gain (real or predicted) for every single candidate. Our goal is to increase guidance for funding strategies.
Current standard of Care
Please go to Treat-NMD and read the two-part article, The analysis and operations of Duchenne Muscular Dystrophy, released in Lancet Neurology, January and February 2010. Summary: 84 international authorities consider over 70,000 medical scenarios and provide a consensus recommendation for operations of DMD. A family version of this research article can be found at Treat-NMD and PPMD. I encourage examining the “printer-friendly” version.
1. Prednisone (glucocorticoid, anti-inflammatory) – considered very first line until pre-existing weight and/or behavioral concerns favor deflazacort.
2. Deflazacort (glucocorticoid) – consider as very first line if favored by pre-existing conditions
1. Ramipril (ACE inhibitor) vs Carvedilol (beta blocker) – check of cardioprotective therapy and worth of CMR (cardiac magnetic resonance).
Trial ID NCT00819845
2. Carvedilol (beta blocker) – preventive efficacy of carvedilol on cardiac dysfunction, prevention of minor damage, measure plasma cTnI
Trial ID NCT00606775
1. Idebenone, Cantena (antioxidant) – compact molecule, antioxidant, 2007 phase II outcome showed improvement of certain cardiac and respiratory measurements
Trial ID NCT01027884 – Santhera Pharmaceuticals
2. GSK2402968 (exon-skipping AO) – exon 51 skipping antisense oligonucleotide, subcutaneous 6mg/kg/week.
Trial ID NCT01254019 – GlaxoSmithKline
3. PTC124, Ataluren (nonsense suppressor) – induces read-through of nonsense (stop codon) mutations. This trial is for nonsense mediated Duchenne and Becker Muscular Duystrophy sufferers that were previously taken care of with Ataluren in a prior PTC-sponsored trial.
Trial ID NCT01247207 – PTC Therapeutics
4. Mechanical insufflation-exsufflation, MI-E (mechanical device) – respriatory physiotherapy, intent is to lower the number of intubations for sufferers with acute respiratory exacerbation.
Trial ID NCT00839033
5. CoQ10 (antioxidant) and Prednisone (glucocorticoid, anti-inflammatory) in Non-Ambulatory DMD – estimated trial completion of December 2010, i am looking forward to the data
Trial ID NCT00308113, terminated
6. Glutamine (amino acid) and Creatine Monohydrate (amino acid) – finished in 2005, since it is not part of standard care neither in phase IV trial, I suppose the data wasn’t positive, will want to examine data.
Trial ID NCT00018109, completed
Phase II / III
1. Sunphenon Epigallocatechin gallate, EGCg (antioxidant, anti-inflammatory) – the important polyphenol in eco-friendly tea, antimutagen, steroid 5 alpha-reductase inhibitor, and considered an anti-inflammatory and antioxidant. find more information at NLM DIP and PubMed: EGCg + Duchenne. once the mdx mouse design was taken care of with EGCg, it led to reduced dietary fiber necroses, reduced proliferation of cartilage in muscle, and enhanced medical symptoms.
Trial ID NCT01183767
2. Creatine vs Glutamine vs Placebo in steroid-naive DMD – measured strength
Trial ID NCT00016653, completed
3. Coenzyme Q10 (antioxidant) and Lisinopril (ACE inhibitor) – primary final result is myocardial performance index
Trial ID NCT01126697
1. SNT-MC17 (calpain/protease inhibitor) –
2. Myoduor (calpain/protease inhibitor) –
3. Idebenone (antioxidant) – DELPHI Extension- compact molecule, antioxidant
Trial ID NCT00758225, active, not recruiting, Santhera Pharmaceuticals
4. Sildenafil, Revatio
Trial ID NCT01168908
5. Glutamine (amino acid) –
Trial ID NCT00296621
6. ACE-031 (protein therapeutic) – intended to increase muscle strength, inhibits signaling by way of a mobile surface receptor called activin receptor type IIB (ActRIIB).
Trial ID NCT01239758, Acceleron Pharma, see Acceleron and Shire’s project HGT 4510.
7. Coenzyme Q10 ( ) –
Trial ID NCT00033189
8. Oxatomide, tinset ( ) –
Trial ID NCT00033813
Phase I / II
x1. Cyclosporin A (anti-inflammatory) –
2. Pentoxyfillin (anti-inflammatory) – phosphodiesterase inhibitor, improves blood flow, and modulates immune activity, product by Frank’s Pharmacy.
Trial ID NCT00102453 and NCT00243789, both equally completed
3. IGF-1 (peptide growth factor) –
Trial ID NCT01207908
4. AVI-4658 (exon-skipping PMO) – exon 51 skip.
Trial ID NCT00844597
5. PRO044 (exon skipping) – exon 44 skip.
Trial ID NCT01037309
6. MYO-029, Stamulumab ( ) –
Trial ID NCT00104078
1. PRO-051 (Prosensa)
2. Sildenaphil –
3. Albuterol – anti-inflammatory
4. MYO-029 (in phase I trial for different disease) – growth factor
5. MyoDys plasmid DNA – (need to verify)
x6. BMN-195 (small molecule utrophin upregulator) – utrophin is a dystrophin homolog
Trial ID not available, see BioMarin
1. AVI-5038 (AVI)
2. SARM (Galapagos)
3. NFkB inhibitor (CRXX) – anti-inflammatory
4. Biglycan (Tivorsan)
5. AAV Minidystrophin – gene therapy
6. AAV U7 – gene therapy
7. nNOS upregulation – anti-inflammatory
8. REMICADE – anti-inflammatory
9. VASTox – large throughput substance screen
Preclinical (prior to animals)
1. Utrophin upregulator (PTC)
2. Tat-utrophin (U.Minn)
3. grownup Stem cells (U.Minn/U.Leuven)
4. PTC Therapeutics / project Catalyst – large throughput substance screen
5. UCLA – large throughput substance screen
6. Genzyme – large throughput substance screen
Other drugs and dietary supplements at this time considered Unnecessary or Inappropriate in accordance to the standard of care article Above
Note: these can be re-categorized if medical trials demonstrate value
1. Oxandrolone – anabolic steroid
2. Botulinum toxin A – proposed as potential medication or prevention of contractures, lacking security study
3. Creatine – no recommendation was given, but a randomized, managed trial failed to show benefit; discontinue if evidence of renal dysfunction.
4. Coenzyme Q10 –
5. Carnitine –
6. Glutamine – amino acid
7. Arginine – amino acid
8. fish essential oil – anti-inflammatory/antioxidant
9. Vitamine E – anti-inflammatory/antioxidant
10. eco-friendly tea extract – anti-inflammatory/antioxidant
11. Pentoxifylline – proposed as potential disease modifying drug