
(HealthDay News) – The U.S. Food and Drug Administration on Tuesday approved a new drug for people who have a rare, inherited type of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The medication, toferson (Qalsody), targets a mutation in the SOD1-ALS gene. ALS attacks and kills nerve cells that control voluntary muscles. With ALS, patients lose nerve cells that affect chewing, walking, breathing and talking. The muscles weaken, and that leads to paralysis. The newly approved medication is made by the company Biogen, which is based in Cambridge, Mass. The FDA approved the drug based on evidence of a reduction in plasma neurofilament light, a blood-based biomarker of nerve injury and neurodegeneration. It did not show that the drug slowed the disease, the Associated Press reported. Still, “the findings are reasonably likely to predict a clinical benefit in patients,” the FDA said in a statement. “Since SOD1 mutations were first identified as a cause of ALS 30 years ago, the familial ALS community has been searching for genetically targeted treatments,” Jean Swidler, chair of Genetic ALS & FTD: End the Legacy, said in a Biogen news release. “Qalsody offers families who have lost generation after generation in the prime of their life to this devastating disease a therapy targeting the underlying cause of SOD1-ALS. Today marks an important moment in ALS research… read on > read on >