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PUBLISHER: FirstWord Group | PRODUCT CODE: 1355801
PUBLISHER: FirstWord Group | PRODUCT CODE: 1355801
Duchenne Muscular Dystrophy - KOL Insight
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Do KOLs believe PTC Therapeutics' Emflaza will retain its leading position in the face of new steroids and novel therapies? What underpins experts' opinion that Sarepta Therapeutics/Roche's gene therapy Elevidys marks a significant advance for DMD patients? What are KOLs safety concerns about Sarepta Therapeutics' next-generation exon-skipping therapy vesleteplirsen? KOLs critically assess the prospects of launched and pipeline therapies.
In addition to the full report, licensed users have access to the following KOL Bulletins via the Attachments area:
- 23 Aug 2023 KOL Bulletin -- Views on the FDA's accelerated approval of Sarepta Therapeutics/Roche's Elevidys (delandistrogene moxeparvovec-rokl) in DMD
Table of Contents
Executive summary (8)
Treatment algorithm
Research objectives (3)
Marketed therapies (45)
- Steroid treatments (8)
- Emflaza (deflazacort; PTC Therapeutics) (8)
- Exon-skipping treatments (27)
- Exondys 51 (eteplirsen; Sarepta Therapeutics) (9)
- Vyondys 53 (golodirsen; Sarepta Therapeutics) (7)
- Viltepso (viltolarsen; Nippon Shinyaku/NS Pharma) (7)
- Amondys 45 (casimersen; Sarepta Therapeutics) (4)
- Other oral treatments (10)
- Translarna (ataluren; PTC Therapeutics) (10)
Pipeline products (Phase III and Phase II) (111)
- Gene therapies (43)
- Elevidys (SRP-9001/delandistrogene moxeparvovec; Sarepta Therapeutics/Roche) (29)
- Fordadistrogene movaparvovec (PF-06939926; Pfizer) (14)
- Next-generation exon-skipping therapies (15)
- Vesleteplirsen (SRP-5051; Sarepta Therapeutics) (15)
- Next-generation steroid treatments (13)
- Vamorolone (ReveraGen Biopharma/Santhera Pharmaceuticals) (13)
- Other mechanisms (40)
- Pamrevlumab (FibroGen) (10)
- CAP-1002 (Capricor Therapeutics) (11)
- Givinostat (Italfarmaco) (7)
- EDG-5506 (Edgewise Therapeutics) (7)
- ATL1102 (Antisense Therapeutics) (5)
Other early-stage mechanisms (Phase I/II) (36)
- Gene therapies (16)
- RGX-202 (Regenxbio) (8)
- scAAV9.U7.ACCA (Astellas Gene Therapies) (4)
- rAAVrh74.MCK.GALGT2 (Nationwide Children's Hospital/Sarepta Therapeutics) (4)
- Exon-skipping therapies (20)
- DYNE-251 (Dyne Therapeutics) (6)
- SQY51 (SQY Therapeutics) (4)
- WVE-N531 (Wave Life Sciences) (5)
- AOC 1044 (Avidity Biosciences) (5)
Future treatment trends in DMD (10)
- Key insights summary (10)
- Newborn screening offers the potential for earlier patient diagnosis and prompt treatment initiation in DMD (8)
KOL details (3)
- KOLs from the USA (1)
- KOLs from Europe (1)
KOL Bulletins (2)
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