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…
At the end of July 2022, biotechnology company Biogen Inc. shared via news release some exciting updates in regards to its therapy tofersen, an investigational treatment for patients with…
Sometimes, it can be difficult to diagnose amyotrophic lateral sclerosis (ALS), a progressive neurological disease which causes nerve cell (motor neuron) death in the brain, brain stem, and spinal…
Thanks to Amylyx Pharmaceuticals, Canada becomes the first country worldwide to have access to an ALS drug branded Albrioza (originally AMX0035). According to an article in Yahoo News, the drug…
The new FDA-approved oral formulation of Radicava ORS (edaravone) is more convenient than the intravenous formulation, yet the efficacy and composition remain the same. The oral formulation may also be…
According to a story from ALS News Today, around 5,000 people get diagnosed with amyotrophic lateral sclerosis (ALS) annually. In addition, costs of treatment for patients average $250,000 out of…
At the end of March 2022, ALS News Today announced that IFB-088, an experimental treatment for amyotrophic lateral sclerosis (ALS), earned Orphan Drug designation from the FDA. The treatment was…
Scientists at London’s University College and a team of scientists at Stanford University simultaneously made the same discovery about amyotrophic lateral sclerosis (ALS): the genetic variant in the protein UNC13A…
Have you ever heard of an astrocyte? This star-shaped cell is found throughout the nervous system. Normally, healthy astrocytes protect motor neurons from damage or degeneration. But according to Medical…
Many people living in rural areas of the U.S. must drive a long distance to receive their medical treatments. According to Yahoo News, twenty-nine-year-old Leah Stavenhagen, now living and…
Three organizations focused on amytrophic lateral sclerosis (ALS) as well as other rare neurodegenerative diseases, have just celebrated the House of Representatives' passage of the ACT for ALS (H.R.3537/S.1813). The…
WKBW Buffalo recently interviewed ALS patients who are advocating for a bill that would grant access to clinical trial drugs during Phase III and before they go through the…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
An October 2021 report published in the Globe and Newswire carried an announcement by Fabio Chianelli, CEO of the biotech company PharmaTher Holdings. The FDA approved orphan drug status for…
From October 6-7, 2021, researchers and stakeholders descended upon the virtual Northeast Amyotrophic Lateral Sclerosis Meeting to discuss new trends, research, and therapies within the field. According to ALS News…
A heartbreaking announcement in PR Week stated that Lisa Stockman Mauriello had died of ALS in her New Jersey home at age 52 on August 4, 2021. As stated, the…
Genetic mutations are associated with a wide range of disorders or medical conditions. According to ALS News Today, researchers sought to understand the underlying genetics associated with sporadic amyotrophic lateral…
Kenneth Mitchell is an actor who has worked on the "Star Trek" franchise since 2017. He's played the Klingon Kol, the scientist Aurellio, Tenavik and Kol-Sha, alongside two other Klingons.…
An article by Sandra Boodman of the Washington Post recently appeared in the Philadelphia Inquirer describing the long journey to an accurate diagnosis for sixty-nine-year-old novelist Cai Emmons. Cai had…
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