Results 211 to 220 of about 2,481 (285)

Spinal muscular atrophy in an upper-middle-income nation before the advent of reimbursed disease-modifying therapies. [PDF]

BMJ Paediatr Open
Sakpichaisakul K, Katanyuwong K, Intusoma U, Paprad T, Suwanpakdee P, Khongkhatithum C, Sanmaneechai O.   +6 more
europepmc   +1 more source

Long-term CSF responses in adult patients with spinal muscular atrophy type 2 or 3 on treatment with nusinersen. [PDF]

J Neurol
Cebulla G, Hai L, Warnken U, Güngör C, Hoffmann DC, Korporal-Kuhnke M, Wildemann B, Wick W, Kessler T, Weiler M.   +9 more
europepmc   +1 more source

Profiling neuroinflammatory markers and response to nusinersen in paediatric spinal muscular atrophy. [PDF]

Sci Rep
Zhang Q, Hong Y, Brusa C, Scoto M, Cornell N, Patel P, Baranello G, Muntoni F, Zhou H.   +8 more
europepmc   +1 more source

Neurological hospitalisations in childhood cancer survivors treated before 2001: findings from the French Childhood Cancer Survivor Study cohort. [PDF]

BMC Neurol
Rajaonera D, Bejarano-Quisoboni D, Grill J, Allodji RS, Pelletier-Fleury N, Journy N, Boussac M, Doz F, Vu-Bezin G, Zidane M, Schwartz B, Haddy N, Bolle S, El-Fayech C, Dufour C, Diallo I, Schleiermacher G, Fresneau B, de Vathaire F.   +18 more
europepmc   +1 more source

Experimental and clinical tests of FDA-approved kinase inhibitors for the treatment of neurological disorders (update 2024). [PDF]

Explor Drug Sci
Aliashrafzadeh H, Liu D, De Alba S, Akbar I, Lui A, Vanleuven J, Martin R, Wang Z, Liu DZ.   +8 more
europepmc   +1 more source

Real-World Data in Children with Spinal Muscular Atrophy Type 1 on Long-Term Ventilation Receiving Gene Therapy: A Prospective Cohort Study. [PDF]

Adv Respir Med
Alajjuri MA, Abusamra R, Mundada V, Narayan O.   +3 more
europepmc   +1 more source

Acceptability, validity and responsiveness of inertial measurement units for assessing motor recovery after gene therapy in infants with early onset spinal muscular atrophy: a prospective cohort study. [PDF]

J Neuroeng Rehabil
Barrois R, Tervil B, Cacioppo M, Barnerias C, Deladrière E, Leloup-Germa V, Hervé A, Oudre L, Ricard D, Vidal PP, Vayatis N, Roy SQ, Brochard S, Gitiaux C, Desguerre I.   +14 more
europepmc   +1 more source

Motor pool selectivity of neuromuscular degeneration in type I spinal muscular atrophy is conserved between human and mouse. [PDF]

Hum Mol Genet
Lee JC, Chung WK, Pisapia DJ, Henderson CE.   +3 more
europepmc   +1 more source

Multidisciplinary physical rehabilitation program of individuals with spinal muscular atrophy in an inclusive school setting. [PDF]

J Pediatr Rehabil Med
Ahmed F, Islam A, Akter S, Al Zubayer MA, Mahmud MN, Yeasmin H, Mawa Z.   +6 more
europepmc   +1 more source

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Chronic childhood spinal muscular atrophies in Algeria

Journal of the Neurological Sciences, 1990
This paper describes a genetic study of the chronic spinal muscular atrophies of late infancy and early childhood in Algeria. There were 50 index patients occurring in 44 kindreds and fourteen secondary cases. Genetic and nosological studies indicated that 52% of the patients constitute a genetically homogeneous subgroup with an age of onset between 3 ...
Charles Geronimi, Meriem Tazir
openaire   +4 more sources