FDAapproves drug for rare form of ALS
The FDA has approved the first treatment that takes a genetics-based approach to slowing or stopping the progression of a rare form of ALS, the debilitating and deadly disease for which there is no cure., die within 3 to 5 years of when symptoms appear, usually of respiratory failure. Also known as Lou Gehrig's disease, people with ALS experience muscle twitching and lose the ability to move their limbs, swallow, speak, and breathe.
While the drug was shown to impact the chemical process in the body linked to degeneration, there was no significant change in people's symptoms during the first 28 weeks that they took the drug, Biogen said in a . But the company noted that some patients did see improved functioning after starting treatment.
Qalsody is given to people via a spinal injection, with an initial course of three injections every 2 weeks. People then get the injection once every 28 days. In trials, 147 people received either Qalsody or a placebo, and the treatment significantly reduced the level of a protein in people's blood that is associated with the loss of control of voluntary muscles.
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