As there is great change in management of muscular dystrophy, due to the fact that many therapies recently became FDA approved (DMD-SMA-ALS) and more in the stage of clinical trials which can be in reach in the near future.
A well-established national patient registry is the starting point for better management to our patients.
The Registry, collates the patients clinical and genetic mutation data to improve the care of DMD, SMA, LGMD, GNE myopathy and other NMD patients in Egypt and the countries in Africa, and to accelerate the best practice for our patients.
This collaboration does not end with the establishments of the Registry – it will continue on through the long term commitment of patient support through awareness campaigns to spread the importance of diagnosis and teaching the standards of care, and to continue to promote the opportunity for patients wanting therapies, of clinicians who keep their clinical information current and provide information to their patients about the existence of new treatment options, and finally, of therapy sponsors to make feasibility enquiries of the Registry and to notify clinicians, through the Registry, of new therapies.