- ISBN: 9788375631128
- Format: A5
- Objętość: 104 strony
SPINAL MUSCULAR ATROPHY: ON THE EVE OF THE CURE
Several novel therapeutic interventions are now in the planning stages for clinical trials, showing that there remains optimism that an effecti ve treatment will eventually be found for SMA.
- ISBN: 9788375631128
- Format: A5
- Objętość: 104 strony
30,00zł
Despite the lack of positive results of trials to date and the significant methodological and scientific challenges to the implementation of SMA trials, SMA remains an attractive disease area for drug development. Although motor disability caused by the loss of motor neurons burdens people with SMA, they maintain normal cognition and, in its milder forms, normal or near normal life expectancy. There have been significant pre-clinical advances over the last decade, so that it is hoped that a successful treatment for SMA is on the horizon. Although trials have not yet resulted in a new treatment for SMA, they have yielded important data for the planning of future trials.
Also, the increasing sophistication and organization of patient groups nationally and internationally has spurred investigator collaboration and the development of clinical trial infrastructure for SMA. Several novel therapeutic interventions are now in the planning stages for clinical trials, showing that there remains optimism that an effecti ve treatment will eventually be found for SMA.
Preface
Chapter 1. A molecular pathomechanism of spinal muscular atrophy
Chapter 2. The epidemiology of spinal muscular atrophy
Chapter 3. Recognition of Proximal Spinal and Bulbar Muscular Atrophy
Chapter 4. Orthopaedic care of a child with spinal muscular atrophy
Chapter 5. Clinical Trial Strategies for Spinal Muscular Atrophy
Chapter 6. From allele segregation to motor neuron death: the long way to unravelling spinal muscular atrophy developmental pathomechanisms
Chapter 7. Spinal muscular atrophy with respiratory distress type 1 (SMARD1)
Chapter 8. Getting to know SMA: the Warsaw group’s experience
Chapter 9. The importance of physiotherapy to children with spinal muscular atrophy (SMA)
