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Academic Journal

General movements in spinal muscular atrophy type 1.

Subjects: MOTOR ability; DISABILITIES; DATA analysis

  • Source: Physiotherapy Theory & Practice; Jun2024, Vol. 40 Issue 6, p1249-1255, 7p

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Academic Journal

Antisense Oligonucleotides (ASOs) in Motor Neuron Diseases: A Road to Cure in Light and Shade.

Subjects: MOTOR neuron diseases; SPINAL muscular atrophy; AMYOTROPHIC lateral sclerosisUNITED States. Food & Drug Administration

  • Source: International Journal of Molecular Sciences; May2024, Vol. 25 Issue 9, p4809, 25p

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Academic Journal

Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care.

Subjects: GENE therapy; SPINAL muscular atrophy; DUCHENNE muscular dystrophy

  • Source: BioTech; Mar2024, Vol. 13 Issue 1, p1, 31p

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Academic Journal

Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape.

Subjects: SPINAL muscular atrophy; GENE therapy; MUSCULAR atrophy

  • Source: International Journal of Molecular Sciences; Sep2023, Vol. 24 Issue 18, p13743, 21p

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Academic Journal

New therapies for spinal muscular atrophy: where we stand and what is next.

Subjects: SPINAL muscular atrophy; NATURAL history; DRUG approval

  • Source: European Journal of Pediatrics; Jul2023, Vol. 182 Issue 7, p2935-2942, 8p, 2 Diagrams

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Academic Journal

Caregivers' Expectations on Possible Functional Changes following Disease-Modifying Treatment in Type II and III Spinal Muscular Atrophy: A Comparative Study.

Subjects: SPINAL muscular atrophy; CAREGIVER attitudes; CAREGIVERS

  • Source: Journal of Clinical Medicine; Jul2023, Vol. 12 Issue 13, p4183, 14p

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Academic Journal

Reproductive genetic carrier screening for cystic fibrosis, fragile X syndrome and spinal muscular atrophy: patterns of community and healthcare provider participation in a Victorian screening program.

Subjects: DIAGNOSIS of fragile X syndrome; CYSTIC fibrosis diagnosis; MATERNAL health servicesSOUTH Australia; NEW Zealand; QUEENSLAND

  • Source: Australian Journal of Primary Health; 2022, Vol. 28 Issue 6, p580-587, 8p

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Academic Journal

The Cure SMA Clinical Trial Experience Survey: A Study of Trial Participant Perspectives on Clinical Trial Management and Patient-Centric Management Practices.

Subjects: CLINICAL trials; SPINAL muscular atrophy; PATIENTS' attitudes

  • Source: Neurology & Therapy; Sep2022, Vol. 11 Issue 3, p1167-1181, 15p

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Academic Journal

The Advent of Omics Sciences in Clinical Trials of Motor Neuron Diseases.

Subjects: MOTOR neuron diseases; CLINICAL trials; DRUG efficacy

  • Source: Journal of Personalized Medicine; May2022, Vol. 12 Issue 5, p758-758, 14p

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Academic Journal

Sharing Clinical Trial Results with Pediatric Participants.

Subjects: CLINICAL trials; CHILD patients; YOUNG adults

  • Source: Applied Clinical Trials; 5/1/2022, Vol. 31 Issue 5, p18-22, 5p

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  • 1-10 of  124 results for ""SPINAL muscular atrophy""