PatientsHigh
We survived. Now we are tired.
“Type 1 parents who got Zolgensma in 2019 are asking about fatigue and school, not survival curves.”
Next product story is function and energy, not CHOP-INTEND.
Neuromuscular · Rare · teaser
Updated August 2026 · SMN1 (5q)
Teaser case. Selected price and peak-sales fields are held for a paid Brief — the rest is the method.
Influencer map
Weighted by trial leadership, guideline voice and advocacy reach — not follower count.
Richard Finkel
Paediatric neurology · St. Jude / Nemours
US
Type 1 · NBS · Trials
Eugenio Mercuri
Outcome measures · Gemelli
IT
Function · EU practice
Laurent Servais
NBS / digital · Oxford
UK
Screening · Endpoints
Thomas Crawford
Adult + paediatric · Johns Hopkins
US
Adults · SOC
Centres & networks
Where the patients already are. Trial-active sites first.
Boston Children's
Boston, US · Cure SMA Care Center
Gemelli
Rome, IT · ERN EURO-NMD
Great Ormond Street
London, UK · NHS highly specialised
Necker
Paris, FR · FILNEMUS
Voice
Thematic synthesis from advocacy, open caregiver forums, X, and clinician social. Directional — not a consented panel. We do not scrape closed groups.
“Type 1 parents who got Zolgensma in 2019 are asking about fatigue and school, not survival curves.”
Next product story is function and energy, not CHOP-INTEND.
“Families will not come back to IT unless there is a clear incremental reason.”
Spinraza defence must be efficacy or a new setting, not loyalty.
“The registry is still full of people the launch campaigns forgot.”
Prevalent Type 3/4 is a commercial pool if someone builds the channel.