Following long-term outcomes of spinal muscular atrophy patients with or without treatment
Observes how spinal muscular atrophy progresses in patients, with or without disease-modifying treatments.
Plain-language title generated from the registry record
Registry title: Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
Key facts
- Where
- Genova, Messina, Milan, and Rome, ItalySee the 5 sites
Where in Italy
- Istituto GasliniGenova, ItalyTaking people
- Nemo SudMessina, ItalyTaking people
- Centro Clinico NemoMilan, ItalyTaking people
- Ospedale Bambino gesuRome, ItalyTaking people
- Policlinico GemelliRome, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Trial code
- NCT05768048
- Record updated
- 14 March 2023
- Original description
This is an observational multicenter retrospective and prospective study on natural history of SMA also considering the 'new natural history' secondary to the availability of commercially available therapies.Read full original descriptionShow less
SMA Natural History Study Evaluating Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
This is an observational multicenter retrospective and prospective study on natural history of SMA also considering the 'new natural history' secondary to the availability of commercially available therapies.
All the patients enrolled to date in the Italian registry, if not part of clinical trials, will be included in the present study.
Inclusion: all patients with mutationsin the SMAN1 gene.
Exclusion: unable to proviude consent.