Therapy and treatment
of ALS and other MN diseases

To date, a causal or disease modifying treatment with strong clinically meaningful effects for the sporadic form of ALS are not available.

In a small number of patients, however, genetic mutations are responsible for the onset and progression of the disease. For these genetically determined ALS variants promising therapeutic options are emerging. Causative therapies for SMA have been available for several years with the drugs nusinersen, risdiplam, and onasemnogen abeparvovec.

Due to the fatal disease course combined with the low incidence, the prevalence of ALS in Germany is very low too (approx. 8000 patients). Hence, neurologists (of which there are also about only 8,000 in Germany) have little experience in the treatment and health care of ALS patients. Treatment of these patients should therefore be carried out in specialized ALS centers. These centers currently treat up to several hundred patients each year.

The possibility of improved health care as well as the implementation of research (both clinical and experimental science, as well as interventional studies) is a decisive advantage in specialized ALS centers. The experience of physicians can be critical in making the diagnosis of ALS, especially for less common phenotypes.

Deep classification of ALS types and ALS variants is becoming increasingly important both for prognosis and in the context of trials. Other advantages of concentrating patients in special centers include rapid recruitment of patients to trials and the possibility of establishing biobanks, a combination of a database and the collection of biological samples (blood, cerebrospinal fluid, tissue).

Unfortunately, such outpatient clinics cannot cover their costs and are therefore dependent on support. Most of the centers therefore offer the possibility to donate specifically and regionally (for further information please refer to the homepage of the respective centers).