Since the cause cannot be removed, the therapy of NMB disease is symptomatic and preventive: existing symptoms are alleviated and complications are prevented.
Rehabilitation methods are not specific to individual diagnoses but are primarily directed towards functional deficits, so rehabilitation does not change the pathophysiological course of the disease, but affects the functional capabilities of the patient. However, there are specificities and fundamental similarities within certain basic groups of diseases, so a good knowledge of these pathophysiological and clinical specificities of individual forms of NMB is necessary for the adequate implementation of health care.
The main form of treatment in the early stages of the disease is regular physical activity with passive stretching of the affected muscles and joints in order to prevent painful shortening of the joints and prolong the period during which the patient can walk.
If joint stiffness occurs, orthopedic surgery can provide relief and improve function. Leg braces can help with independent standing and walking when hip and leg muscles are so weakened that this is impossible.
A person who can no longer stand should be assisted by a suitable wheelchair. Sitting upright in bed is necessary to prevent fluid from accumulating in the lungs. If scoliosis develops, external braces can be placed to prevent its progression, or surgery can be performed.
There are three components in the complex rehabilitation of persons with NMB: medical, psychological and social. Ideally, they should be coordinated and implemented in a coordinated way, which should lead to the best possible cooperation of the rehabilitation team so that ultimately the sick person has adequate care.
In the past few years, there have been the first concrete advances in the development of a drug for certain forms of NMB. First, 2014 was marked by the appearance of Ataluren, the first drug for Duchenne muscular dystrophy, but a drug intended for only one group of specifically occurring gene mutations that manages to slow down the progression of the disease to a certain extent, while in 2017, Spinraza (nusinersen) was approved in the European Union, the first drug for spinal muscular atrophy, which shows unprecedented positive results and gives patients hope not only in slowing down the disease but also in restoring some of their functions. Due to the extremely high price of the drug, Spinraza was only included in the Basic List of Medicines of the Croatian Institute of Health Insurance in April 2018, but only for people under the age of 18 who are not on a ventilator.
