“We are grateful to Cytokinetics for their partnership and transparency, enabling the global research community to leverage these data in their ongoing analyses and research activities,” said Neil Thakur, Ph.D., Chief Mission Officer at The ALS Association.

We filed comments with the FDA asking for clarity and heightened urgency in the agency’s Action Plan for Rare Degenerative Diseases, including ALS.

When the FDA recently accepted Biogen’s new drug application for tofersen, it signaled hope for thousands of people with SOD1 mutations that cause ALS. The FDA has granted priority review to this new drug application and is expected to decide on whether or not to approve tofersen by January 25, 2023.

The researchers focus on an earlier phase of ALS development, which is commonly understood to be a “silent” phase – before symptoms of the disease begin to manifest. Those minor motor impairments in the earlier phase are currently insufficient for a confirmed diagnosis.

The ALS Association filed formal objections with the Institute for Clinical and Economic Review, commonly known as ICER, over their flawed draft report on the cost-effectiveness of AMX0035 and oral edaravone.

This morning we joined other ALS organizations releasing an open letter urging the FDA to quickly approve AMX0035.

Today, ICER (the Institute of Clinical and Economic Review) issued its initial draft report on the cost-effectiveness of AMX0035 and oral edaravone.

AMX0035 is first treatment funded by ALS Ice Bucket Challenge donations to be considered for approval by FDA.

People with ALS, caregivers and people at risk of an ALS diagnosis have the opportunity to help the National Institute of Neurological Disorders and Stroke (NINDS) review applications for funding to support research into expanded access.

The Food and Drug Administration has extended the timeline of its review of AMX0035 citing additional data further showing that it is effective at reducing harms associated with the disease.

Follow-up analyses from the Phase 3 Valor study and the open-label extension study of tofersen, an investigational antisense drug, showed that the drug was effective in slowing down progression of ALS in people with SOD1 mutations. 

We submitted a letter to the FDA signed by 37 clinicians who specialize in treating people with ALS calling on the FDA to swiftly approve AMX0035.

Every year, hundreds of people with ALS, their caregivers, friends and family come together to discuss progress in the fight against ALS and how we advance ALS research, accelerate the drug development process and make it possible for people with ALS to live their lives as they want by engaging policymakers to support the ALS community. Registration is now open for our annual advocacy conference, which will be held June 14 -16 from 3:00-4:30 p.m. ET. Due to visitor restrictions at the U.S. Capitol Complex, the event will be virtual again this year.

New long-term analysis published in the Journal of Neurology, Neurosurgery & Psychiatry showed AMX0035 was effective at reducing a variety of harms associated with ALS during the previously published Phase 2 Centaur trial.

We welcome the FDA’s approval Thursday of an oral formulation of the drug edaravone, produced by Mitsubishi Tanabe Pharma America (MTPA) to treat ALS.

People living with ALS and adult caregivers can help researchers learn more about stigma and quality of life in ALS.

The ALS Association, in collaboration with ALS Finding a Cure, recently awarded $400,000 to support research into the role SARM1 gene mutations play in the development and progression of ALS, with the ultimate goal of using an improved understanding of SARM1 to develop new treatments.

The ALS Association has launched a new funding opportunity to support exploratory research that has the potential for a significant impact on the fight against ALS.

Under the legislation, Minnesota will disperse $20 million to the Minnesota Officer of Higher Education to award competitive research grants to scientists studying the prevention, treatment, causes or cures of ALS. Applicants may range from research facilities, universities or health systems located in Minnesota. An additional $5 million will be dedicated to helping caregivers care for loved ones living with ALS. These caregiving funds will be appropriated to the Minnesota Department of Aging to fund local organizations dedicated to providing caregiver support programs that serve Minnesotans in their homes and communities. The funds may also be used to provide respite care. 

Earlier today the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6 to 4 against recommending AMX0035 for approval to treat people with ALS. It’s important to note that the Advisory Committee’s views are not binding on the FDA. Following the vote, the ALS Association called on the FDA to take into account the strong safety profile of AMX0035, as well as the serious unmet medical need of people living with this devastating condition, and approve the drug for clinical use.