Scientific Projects from the SMArtCARE Network
The academic network around SMArtCARE aims to evaluate the long-term course of patients under therapy from a scientific point of view in order to gain additional knowledge about SMA, and the safety and the benefit of the drugs compared to other therapies.
Therefore, over the entire duration of the SMArtCARE data collection, the results obtained will be statistically evaluated and published in scientific journals. In these publications, no personally identifiable data (such as names or characteristics) will be published from which conclusions can be drawn about you or your child.
The SMArtCARE data are currently being used to address the following research questions:
Factors influencing respiratory involvement in spinal muscular atrophy types I through III
Dr. M. Groß, Prof. Summ, Dr. Grimm, Dr. Otto-Sobotka
Natural History in SMA - Time to loss of best ever reached motor milestone
Dr. Hanna Sophie Lapp
SMA Scoliosis - Developing a Data-Driven Scoliosis Progression Model in 5q-Spinal Muscular Atrophy in the (Gene)-Therapy Era
Dr. Claudia Weiß, Dr. Tu-Lan Vu-Han
Parenthood, family planning, and pregnancy experience and outcomes in patients with Spinal Muscular Atrophy
Prof. Dr. Maggie Walter, Simone Thiele
Impact of SMN2-augmenting therapies on the hospitalization rate of adult patients with spinal muscular atrophy
Dr. Benjamin Stolte, Prof. Dr. Tim Hagenacke
Outcome in severe cases of SMA with prenatal or neonatal onset - international data collection
Prof. Dr. Kirschner
Comparative analysis of data from patients on therapy with nusinersen or onasemnogen-abeparvovec and the influence of a change in therapy
PD Dr. Astrid Pechmann, Prof. Dr. Janbernd Kirschner in cooperation with the Institute for Medical Biometry and Statistics (IMBI) of the University Medical Centre Freiburg
Evaluation of Functional Outcomes in Patients with 5q-Associated Spinal Muscular Atrophy Following a Switch from Nusinersen to Risdiplam Compared to Long-Term Nusinersen Therapy (Type II and Type III),
Dr. Deike Weiss, Dr. Jessika, Johannsen, PD Dr. Jonas Denecke
SMA Newborn Screening in Germany: Long-Term Course in Preterm Infants with SMA,
Prof. Dr. Regina Trollmann
Characterization of Patients with Late-Onset SMA (Type 4) and Asymptomatic Adult Biallelic SMN1 Carriers – Identification of Disease Modifiers and Biomarkers for Predicting Disease Course in SMA,
Prof. Dr. Marcus Deschauer
Assessment of cognitive and language development in SMA patients with up to 4 copies of the SMN2 gene who were treated in routine clinical practice with SMN-modifying therapy or gene-addition therapy,
Dr. Jessika, Johannsen
Is microcephaly a newly recognized phenotype of the central nervous system (CNS) in pediatric SMA patients?,
Dr. Jessika, Johannsen
A registry-based longitudinal comparative analysis of the efficacy of nusinersen versus risdiplam in adult patients with spinal muscular atrophy over a 30-month period,
Prof. Dr. Maggie Walter, Prof. Dr. Hagenacker
Impact of SMN2-augmenting therapies on the hospitalization rate of adult patients with spinal muscular atrophy
Dr. Benjamin Stolte, Prof. Dr. Tim Hagenacke