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…
An announcement via PR NewsWire by NeuroSense Therapeutics based in Cambridge, Massachusetts carried the results of its multi-dose study of PrimeC to treat amyotrophic lateral sclerosis (ALS) Expectations are…
According to a story from CNN, a new treatment was recently approved by the US Food and Drug Administration (FDA) for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's…
The loss of axons (nervous system wiring) has been responsible for various neurodegenerative diseases such as peripheral neuropathies, Parkinson’s disease, glaucoma, and amyotrophic lateral sclerosis (ALS). According to a…
It can be extremely difficult to incentivize drug development for rare diseases or conditions. To combat this, decades ago, the FDA created the Orphan Drug Act. Now, Orphan Drug designation…
Verge Genomics, based in San Francisco is one of a series of biotechs that have entered into a new era using AI in drug discovery. The company is backed by…
An article in BioSpace announced that the FDA’s CNS committee met twice in recent months to discuss the merits of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). Within…
StatNews recently carried an article describing a paper prepared by New York’s Tisch Research Center and published this week in the Brain Communications journal. The paper contains promising breakthroughs on…
According to a story from Upstate Medical University, the college's ALS Treatment and Research Center has been a certified center of excellence for research and clinical care that has helped…
According to a story from Market Screener, the clinical stage biopharmaceutical company Kringle Pharma, Inc., has recently announced an update on the progress of its phase 2 clinical trial. The…
According to a story from Medical Xpress, a medicine used to treat high blood pressure and enlarged prostate could be a potential therapy for amyotrophic lateral sclerosis (ALS), a rare…
© Copyright Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
