The newly-approved drug, Risdiplam, is the first registered drug for Spinal Muscular Atrophy in Egypt.
The Egyptian National Drug Authority has just approved the first drug in the country to treat spinal muscular atrophy (SMA), a rare disease which affects one in every 10,000 people.
SMA is a genetic disorder that causes the muscles to waste away due to loss of specialized nerve cells. Eventually, it results in chronic pain and complete loss of movement. People with the disease are born with it, and see their symptoms slowly worsen with age. To this day, there are few treatments for the disease.
The newly-approved drug, Risdiplam, is the first registered drug for the disease in Egypt. It is largely used to relieve pain and other symptoms.
The news comes closely after the proliferation of the #Save_Rasheed social media campaign, which hopes to raise USD 2.1 million to save Rasheed Yasser Taha, a toddler with SMA. The fund is meant to purchase Zolgensma, a USD 2.1 million drug that young SMA patients must take before the age of two, or else they will lose all ability to move for the rest of their life. The campaign is still on-going, and is accepting donations through the ‘Save Rasheed’ group on Facebook.