We talked with Dr. Ahmad Al Khleifat, recent winner of the distinguished ENCALS Young Investigator Award and postdoctoral fellow from King’s College London, to learn more about his research focused on disease gene identification through next generation sequencing, coupled with advanced data analysis to deliver diagnostic tools for complex disease genetics.
Expanded Access, or “compassionate use” as it is often referred, allows patients with a terminal diagnosis early access to new therapeutics that show promise – even if the patient is not involved in the ongoing clinical trial – or if the medication has not yet been approved by the FDA.
The American Academy of Neurology, The ALS Association and the American Brain Foundation have awarded the 2022 Sheila Essey Award to Matthew Kiernan of the Brain and Mind Centre at the University of Sydney in Australia and a member of the American Academy of Neurology. We recently caught up with Dr. Kiernan on Connecting ALS to discuss advances in ALS research since he first entered the field and how the Essey Award will help move his research forward.
The ALS Association has launched a petition calling on public and private health insurers, as well as federal and state governments to prohibit the use of arbitrary, discriminatory value assessments that limit access to ALS drugs.
Carianne “Cari” Meystrik is a true hero to her family, friends and the entire staff at The ALS Association Tennessee Chapter. Cari and her husband Chris had only been married for 6 ½ years when she was diagnosed with ALS in 1998 while pregnant with her fourth child. The couple thought their dreams of growing old together and raising a family were over. Instead, despite the various obstacles brought on by ALS, the Meystrik family has had incredible adventures and made precious memories that carry them through the tough times.
The ALS Association has awarded $3.6 million to four interventional trials through its new Clinical Trial Awards program. The Clinical Trial Awards program is open to industry and academic investigators proposing novel or repositioning approaches for ALS.
Each year on February 11, the world celebrates International Day of Women and Girls in Science Day. This year we’re shining a spotlight on Dr. Allison Ebert, Ph.D., Leadership Team Member at The ALS Association Wisconsin Chapter & professor at the Medical College of Wisconsin. Dr. Ebert breaks down the challenges of finding effective therapies for people living with ALS and the important work happening in her lab.
The National Institute of Neurological Disorders and Stroke (NINDS) issued a request for input recently to help guide its work supporting ALS research. The ALS Association is submitting input telling the agency that speed matters. We are encouraging NINDS and the rest of NIH to focus to find ways to use research to advance the health of people with ALS as quickly as possible.
The Institute of Clinical and Economic Review, commonly known as ICER, has opened a review of AMX0035 to determine the cost-effectiveness of the drug. The ALS Association is committed to making sure ICER’s review does not discriminate against people with ALS and that its analysis does not prevent people with ALS from accessing promising treatments.
Leadership Team Member & Professor at the Medical College of Wisconsin, Dr. Ebert breaks down the challenges of finding effective therapies for ALS and how you can support the important work happening in her lab.
We talked with Dr. Yuka Koike, postdoctoral fellow working under her mentor, Dr. Leonard Petrucelli in the Petrucelli lab at Mayo Clinic Jacksonville, to learn more about her and her ALS research focused on revealing how TDP-43 mediated RNA dysfunction contributes to ALS/FTD disease.
The Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database recently received the Healey Center International Prize for Innovation in ALS, a $50,000 prize. The database is the largest collection of ALS clinical trial data and includes de-identified records of 11,000 patients from 23 clinical trials.
People with ALS and their caregivers who participated in a recent ALS Focus survey said that telehealth appointments improved their quality of life by saving them time and money, and that the appointments are just as good as in-person health care visits.
Research funded by The ALS Association has found that NFL players are four times more likely to be diagnosed with ALS and die from the disease than people who never played in the league, adding to the mounting evidence of a link between playing football and ALS.
We talked with Dr. Janani Parameswaran, postdoctoral fellow from Dr. Jie Jiang’s lab at Emory University in Atlanta, to learn more about her and her ALS research focused on unraveling the underlying disease mechanism.
We asked the FDA to treat the approval review process of AMX0035 with urgency. Specifically, we sent a letter to FDA asking the agency to conduct a Priority Review of Amylyx’s New Drug Application (NDA) for AMX0035 and then approve it. The Priority Review is an expedited review process, as opposed to the Standard Review process, which can take upwards of a year after the agency accepts submission of the NDA.