Relyvrio, Radicava ORS Cost-effective at Current Prices: ICER
ALS News Today
by Marisa Wexler, MS
3d ago
The recently approved therapies Relyvrio (sodium phenylbutyrate and taurursodiol) and Radicava ORS (edaravone) are both likely to be cost-effective for treating amyotrophic lateral sclerosis (ALS), according to a new analysis from the Institute for Clinical and Economic Review (ICER). The report, “The effectiveness and value of AMX0035 and oral edaravone for amyotrophic lateral sclerosis: A summary from the Institute for Clinical and Economic Review’s Midwest Comparative Effectiveness Public Advisory Council,” was published in the Journal of Managed Care and Specialty Pharmacy. Relyvrio ..read more
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‘Good Enough’ Means Something Different in Life With ALS
ALS News Today
by Kristin Neva
4d ago
In the Jan. 23 episode of “The Happiness Lab” podcast, Simone Stolzoff, author of the book “The Good Enough Job: Reclaiming Life from Work,” argues that we should stop hunting for the perfect job. Stolzoff and podcast host Laurie Santos, a psychology professor, discuss how society’s view of work has changed over the course of a generation. Today, for example, rather than work being simply a way to pay the bills and support a family as it used to be, we’ve made it a major part of our identity. Stolzoff suggests that we should change our mindset to accept that a job may be just “good enough ..read more
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UniQure Acquires Rights to Develop APB-102
ALS News Today
by Teresa Carvalho, MS
4d ago
uniQure has entered an agreement to acquire the full rights to Apic Bio‘s investigational gene therapy APB-102, which is being developed for amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations. uniQure now is planning to start a Phase 1/2 clinical trial of APB-102 later this year. Under the agreement, Apic Bio will receive an upfront payment of $10 million, and is entitled to up to $45 million in additional payments if certain regulatory milestones are met, and royalties for any sales. “I am very proud of the contributions Apic Bio has made to bring APB-102 to the cusp of ..read more
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New Pig Model May Better Capture ALS at Its Earliest Stages
ALS News Today
by Steve Bryson, PhD
4d ago
A new pig model of amyotrophic lateral sclerosis (ALS) more accurately reflects the gradual motor neuron loss and disease progression seen in patients, and it may speed the development of ALS treatments, included gene and cell-based therapies, researchers report. The model carries a mutation in the SOD1 gene, an underlying cause of familial ALS, and it has a longer presymptomatic period than ALS mouse models, which are marked by rapid disease progression. Examined before the onset of motor symptoms, the pig model shared many features with patients and mouse models, such as damage to motor neu ..read more
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Phase 1 Safety Study Clears Path to Test Utreloxastat in ALS Patients
ALS News Today
by Steve Bryson, PhD
4d ago
Utreloxastat (PTC857), an investigational therapy for people with amyotrophic lateral sclerosis (ALS), was safe, well tolerated, and displayed promising pharmacological properties in healthy people, according to a first-in-human Phase 1 study. Based on these findings, the therapy’s developer, PTC Therapeutics, has launched a Phase 2 trial (NCT05349721) to evaluate it in up to 258 ALS adults, ages 18–80. Recruitment is underway at sites in California, Florida, Kansas, and Texas. Results from the Phase 1 trial were shared in the study, “First-in-Human Studies of Pharmacokinetics and Safety of U ..read more
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5 Things I’d Tell My Pre-ALS Self That I Learned From Caregiving
ALS News Today
by Juliet Taylor
4d ago
I’ve read dozens, if not more, of the kind of column I’m about to write: first-person narratives that offer advice to one’s younger self, full of lessons learned from difficult life experiences. Before my late husband, Jeff, was diagnosed with ALS, I devoured these columns, always looking for some relatable wisdom I could use to better find my way in the world. Jeff’s diagnosis changed me so much that I now divide my life into two distinct periods: before and after ALS. Once ALS hit our family, all of my collected quotations and inspiring phrases went out the window. My go-to phrase in early ..read more
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Higher ‘Good’ Cholesterol Linked to Worse ALS Survival Rate
ALS News Today
by Patricia Inácio, PhD
4d ago
Elevated levels of high-density lipoprotein (HDL) — commonly called the “good” cholesterol — are significantly associated with a poorer survival rate among people with amyotrophic lateral sclerosis (ALS), according to a population-based study in the Netherlands. In contrast, levels of total cholesterol and low-density lipoprotein — the “bad” cholesterol — were lower in people with more advanced disease stages. The findings suggest that lipid, or fat molecules “may contain valuable information about disease severity and prognosis, because [blood] concentrations seem to be dependent on disease ..read more
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Watching a Film About Robots on Mars Through the Lens of ALS
ALS News Today
by Kristin Neva
1w ago
Now that my husband, Todd, is paralyzed and uses a noninvasive ventilator, leaving the house is too hard on him. So when we have a date night, we watch a movie at home. Last weekend, we watched “Good Night Oppy,” a documentary about twin rovers that NASA sent to Mars to find evidence of past water on the planet. They named the rovers Spirit and Opportunity, and from early in their development, each seemed to take on her own personality. Spirit was plagued with technical failures, but Opportunity passed every test with flying colors. Oppy was the lucky one. Spirit vanished after landing; the m ..read more
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FDA Seeks Input on Tofersen’s Approval for ALS
ALS News Today
by Steve Bryson, PhD
1w ago
The U.S. Food and Drug Administration (FDA) has announced an advisory committee meeting seeking advice on whether to approve Biogen‘s investigational therapy tofersen for the treatment of amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The public meeting, to be held online March 22, by the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, will determine whether tofersen can be approved based on the strength of biomarker data from the VALOR Phase 1/2/3 trial (NCT02623699). The FDA began reviewing the treatment in June under its accelerated approval pathwa ..read more
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Amylyx Selects Neopharm to Market AMX0035 for ALS in Middle East
ALS News Today
by Teresa Carvalho, MS
1w ago
Neopharm has acquired exclusive rights to commercialize AMX0035 (sodium phenylbutyrate and taurursodiol) in Israel, Gaza, the West Bank, and to the Palestinian Authority as a treatment of amyotrophic lateral sclerosis (ALS). The company’s agreement with Amylyx Pharmaceuticals makes it responsible for the distribution and marketing of AMX0035 in these Middle East regions, pending its approval by regulatory authorities. Neopharm also will be in charge of the filings and duties required for regulatory review and therapy reimbursement. “This agreement with Neopharm is an important step ..read more
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