Jean's life story is one of extraordinary resilience, a testament to the strength of the human spirit in the face of rare and challenging medical conditions. Born in 1943 in…
What do progressive supranuclear palsy (PSP), frontotemporal dementia (FTD), and amyotrophic lateral sclerosis (ALS) all have in common? People with these conditions often face long diagnostic delays due to challenges…
The ALS space looked promising in 2022 after Relyvrio’s approval and a green light for Oalsody in 2023. Then progress stalled a bit in 2024 as a result of Relyvrio…
An American triathlete, Jon Blais, received a diagnosis of amyotrophic lateral sclerosis (ALS) in 2005 when he was 33 years old. The disease is incurable. Jon was told he had…
According to a recent article in MedicalXpress, scientists have discovered a possible method of treating amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The study was also published in Science…
Historically, it has been difficult to incentivize companies to develop therapies for rare or “orphan” conditions. The National Conference of State Legislatures (NCSL) explains that “orphan” conditions are: neglected conditions…
The European Medicines Agency (EMA) explains that monepantel is an anthelmintic therapy, or a type of therapy that is used to expel worm-like parasites called helminths or nematodes from the gut.…
Amyotrophic lateral sclerosis (ALS), ravages nerve cells in the spinal cord and brain. It is at times called Lou Gehrig’s disease, named after the famous baseball player who died of…
Prilenia Therapeutics, Waltham, Massachusetts and Naarden, Netherlands is a biotechnology company with a focus on developing therapeutics to treat neurodevelopmental disorders and neurodegenerative diseases. Prilenia presented data this month at…
In December 2023, clinical-stage biopharmaceutical company Clene Nanomedicine ("Clene") shared that it had met with the U.S. Food and Drug Administration (FDA) regarding Accelerated Approval for CNM-Au8®, an experimental therapy…
Strokes, traumatic brain injuries, amyotrophic lateral sclerosis, Alzheimer's disease, and multiple sclerosis all share something in common: they all have profound effects on the brain and its function. They can…
According to a recent BioSpace article from Heather McKenzie, the U.S. Food and Drug Administration's Cellular, Tissue, and Gene Therapies Advisory Committee recently voted against approving NurOwn, an investigational…
The long-awaited decision by the European committee CHMP was announced recently by Amylyx Pharmaceutical (the Company) based in Cambridge, Massachusetts just as anticipated. The EMA issued its negative opinion of…
According to a recent article in People Magazine, this Illinois family has a dream: to turn amyotrophic lateral sclerosis (ALS) from a fatal to a chronic illness in Brian’s…
According to a story from BioPharma Dive, Apellis Pharmaceuticals will now join the ranks of pharma companies that have seen an investigational therapy being developed for amyotrophic lateral sclerosis falter…
According to a story from PR Newswire, the QurAlis Corporation, a clinical stage biotech company, recently announced the completion of the review of the Clinical Trial Regulation (CTR) in the…
Relyvrio was officially approved by the FDA for treating ALS in September 2022. However, according to an article in BioPharma Dive, there was considerable controversy surrounding the clearance of the…
The FDA estimates that there are about five hundred people in the USA with the SOD1 mutation out of a total of approximately thirty thousand ALS patients. According to a…
Currently, there are no cures for amyotrophic lateral sclerosis (ALS). Symptomatic therapy, alongside disease-modifying treatments like Rilutek, Radicava, and Relyvrio, may be used to manage this condition. However, there…
On March 22, 2023, via PRNewswire, the FDA announced that its CNS Drugs Advisory Committee voted unanimously in favor of a potential accelerated approval of tofersen to treat people with…
STATHMIN-2 (STMN2) is a protein that has been proven to play a role in axonal development, repair, and stability. This protein is highly expressed in human motor neurons; research…
In the United States, Orphan Drug designation is granted to drugs or biologics being developed to treat, prevent, or diagnose a rare disease. These are conditions affecting fewer than…
Two Southern California University studies showed a reduction of neurodegenerative activity in amyotrophic lateral sclerosis (ALS) research models with each using a different treatment route. As reported in Inside Precision…
Doddie Aid, which begins January 1st and continues for the following six weeks, aims to encourage people to get active while raising funds to support research into motor neuron disease…
According to a story from myscience.org, neurodegenerative diseases, such as amyotrophic lateral sclerosis, are diseases that progressively worsen over time. However, progression in this disease is not consistent across all…
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