Neuromuscular · Rare
Updated August 2026 · DMD (dystrophin, Xp21)
Influencer map
Weighted by trial leadership, guideline voice and advocacy reach — not follower count.
Craig McDonald
CINRG / functional endpoints · UC Davis
US
NSAA · Natural history · Trial design
Francesco Muntoni
Neuromuscular lead · UCL / GOSH
UK
Exon skip · EU access · Natural history
Pat Furlong
Advocacy founder · PPMD
US
Access · Family voice · Policy
Eugenio Mercuri
Paediatric neurology · Gemelli, Rome
IT
Outcome measures · EU trials
Jerry R. Mendell
Gene therapy pioneer · Nationwide Children's
US
AAV · Elevidys legacy
Annemieke Aartsma-Rus
Skip science · LUMC Leiden
NL
AON design · Dystrophin quantification
Laurent Servais
Trialist · University of Oxford
UK
Newborn screening · Digital endpoints
Kevin Flanigan
Gene transfer / skip · Nationwide Children's
US
U7 snRNA · Duplications
Volker Straub
John Walton lead · Newcastle
UK
Imaging · ERN-NMD
Elizabeth Vroom
Advocacy · World Duchenne / UPPMD
NL
Global access · Standards of care
Nathalie Goemans
Clinic + trials · UZ Leuven
BE
SOC · European practice
Crystal Proud
High-volume GT centre · CHKD Norfolk
US
Elevidys real world · Safety
Centres & networks
Where the patients already are. Trial-active sites first.
Nationwide Children's Hospital
Columbus, US · PPMD Certified / MDA
Highest GT volume historically
Boston Children's Hospital
Boston, US · PPMD Certified
Children's Hospital of Philadelphia
Philadelphia, US · PPMD Certified
UCLA Neuromuscular
Los Angeles, US · PPMD Certified
UC Davis / CINRG
Sacramento, US · CINRG
Natural history core
Great Ormond Street / UCL
London, UK · ERN EURO-NMD
John Walton Muscular Dystrophy Research Centre
Newcastle, UK · TREAT-NMD / ERN
Fondazione Policlinico Gemelli
Rome, IT · ERN EURO-NMD
Hôpital Necker-Enfants malades
Paris, FR · FILNEMUS
UZ Leuven
Leuven, BE · ERN EURO-NMD
LUMC
Leiden, NL · ERN EURO-NMD
National Center of Neurology and Psychiatry
Tokyo, JP · Remudy registry
Voice
Thematic synthesis from advocacy, open caregiver forums, X, and clinician social. Directional — not a consented panel. We do not scrape closed groups.
“After the liver deaths, Facebook groups flipped from 'when can we get it' to 'how do we even think about this.'”
Consent, centre quality and ambulatory-only labelling will suppress near-term GT uptake more than price will.
“Ports, Monday infusions, school missed — families call it a part-time job for a Western blot.”
Any oligo that is monthly or less, or oral, has a conversion story even at similar dystrophin.
“Weight, fractures, mood — caregivers rank steroid AEs next to the disease itself.”
Agamree's commercial case is quality of SOC, not incremental NSAA.
“US commercial GT vs EU HTA timelines. Same mutation, different life.”
EU5 revenue for premium DMD drugs lags US by years; model it that way.
“We keep boys walking longer. They now die of the heart. Our clinics are not staffed for that.”
Assets with a cardiac endpoint (deramiocel, next-gen constructs) map to how KOLs actually spend their week.
“NSAA misses and biomarker approvals have made European KOLs openly sceptical of the last wave.”
The next BLA needs a functional story a neuromuscular clinic will defend in an MDT.
“Treat earlier or admit the constructs are too weak for established disease.”
NBS expansion is a volume and a timing lever for every GT and oligo model.
“Paediatric centres discharge young men into a void.”
Prevalent non-ambulatory adults are invisible in most sponsor models and most clinic networks.