We urgently need more and better ALS treatments. Because of this, getting promising therapies out of the laboratory and into clinical testing as quickly as possible is key to making ALS a livable disease.
There is an urgent need for new and improved therapies for ALS, as there is still no cure. To help accelerate drug development and get promising treatments one step closer to regulatory evaluation, the ALS Association has committed nearly $4 million to support four early-stage clinical trials through our Hoffman ALS Clinical Trial Awards.
We are excited to share our 2023 Mid-Year Report, detailing the work and achievements that have shaped the first half of this year, touching on all aspects of our mission: research, care services and advocacy.
We talked with Dr. Miriam Linsenmeier, a postdoctoral fellow at the University of Pennsylvania’s Perelman School of Medicine, to learn more about her research, as well as her interests outside the lab.
This last year has seen incredible progress in the fight to create a world without ALS. The FDA approval of Relyvrio (AMX0035) in 2022 and Qalsody (tofersen) last month are significant steps in the effort to make ALS a livable disease. As we celebrate the success of these long-term investments, we continue to fund the next generation of possible treatments, as well as the researchers that are searching for them.
The FDA announced it has granted accelerated approval to tofersen, a treatment for people living with mutations of the SOD1 gene. This is a significant victory for the ALS community and our efforts to make ALS livable for everyone, everywhere, until we can cure it.
The ALS Association commends the FDA for approving tofersen under the agency’s accelerated approval pathway for the treatment of people living with ALS connected to mutations in the SOD1 gene. This marks the first time the FDA has approved a treatment for ALS under accelerated approval and serves as a promising endorsement of the antisense technology that underlies tofersen.
We thank the FDA Advisory Committee for their clear and compelling recommendation that tofersen be approved under the accelerated approval pathway. We urge the FDA to swiftly approve this urgently needed new treatment. Americans living with this rare and aggressive genetic form of ALS cannot wait.
Larry Falivena, member of The ALS Association’s Board of Trustees who is living with ALS, shares his unique experience with the disease and his perspective on genetic testing.
The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and the Northeast ALS (NEALS) Consortium announced topline results from Regimen D of the HEALEY ALS Platform Trial, which is evaluating pridopidine versus placebo in adults with ALS.