Do you love golfing? Interested in making a difference for patients with amyotrophic lateral sclerosis (ALS)? If you're located in or around Oceanside, CA, then I have an event…
In the past, some drug developers were not incentivized to develop treatments for those within the rare disease community, considering the smaller patient size. However, the FDA created the Orphan…
Amyotrophic lateral sclerosis (ALS) is a rare, neurological disorder that is characterized by the progressive death of nerve cells and muscle weakness. As time passes, many affected individuals experience difficulty…
A recent announcement in Biospace by Capsida Biotherapeutics and CRISPR Therapeutics of their newly-formed partnership offers renewed hope for amyotrophic lateral sclerosis (ALS) and Friedreich’s Ataxia patients. The companies believe…
In early June 2021, SPG302, an investigational therapy for patients with amyotrophic lateral sclerosis (ALS), received Orphan Drug designation from the FDA. According to ALS News Today, the treatment…
Normally, amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder, affects individuals between ages 50-60, with prevalence increasing with age. Once diagnosed, patients typically survive for another 3-5 years. However,…
Noémie has four wonderful, healthy children. She has made it clear that amyotrophic lateral sclerosis (ALS) is not the end for her and that she values every precious moment…
Clinical trials are extremely beneficial in learning how new and burgeoning treatment options can benefit patients. At the same time, these trials also offer insight into where treatments may not…
Gene editing, CRISPR specifically, has become much more well-known and heavily used in the recent past. Advances in this field have allowed medical professionals to treat conditions that had seen…
According to a press release from April 8, 2021, a partnership between Project ALS and the ALS Association has allowed for the development of a Phase 3 clinical trial. Two…
A note from Lisa: Please read and share this piece with anyone living with ALS or any life-threatening condition that can benefit from compassionate use: Lisa’s friend of thirty years…
Lisa Stockman Mauriello of Summit, New Jersey, is not alone in her struggle to gain access to the company’s experimental ALS drug, tofersen. In fact, according to a recent article…
In mid-March 2021, Mitsubishi Tanabe Pharma Canada ("MTP-CA") shared that its infusion therapy RADICAVA (edaravone) is now available for some Canadian patients with amyotrophic lateral sclerosis (ALS). The treatment will…
Researchers from Northwestern University have discovered a compound with the ability to stop the progressive degeneration of motor neurons in those with amyotrophic lateral sclerosis (ALS). The continued breakdown of…
A new licensing and supply deal has been made between Mitsubishi Tanabe Pharma America Inc. (MTPA) and Aquestive Therapeutics concerning the commercialization of riluzole in the United States. Riluzole, being…
Recently, clinical stage biotechnology company Prilenia Therapeutics B.V. ("Prilenia") announced that the first patient has been enrolled in a Phase 2/3 clinical trial evaluating pridopidine for patients with amyotrophic…
A recent article in the Encinitas Advocate heralded the dedication of Stan Crooke, n-Lorem Foundation’s CEO and founder. The nonprofit Foundation’s team acts as middlemen, working with patients who…
Target ALS has recently launched a new research initiative, in which they will dedicate $15 million to amyotrophic lateral sclerosis (ALS) research, specifically the discovery of biomarkers. These millions will…
This past month, the Ice Bucket Challenge was brought back into the spotlight when co-founder of the ALS Ice Bucket Challenge, Pat Quinn, unfortunately passed away. As the ALS Association…
According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…
In a recent press release, Catalent and BrainStorm Cell Therapeutics Inc. ("BrainStorm") announced a partnership to manufacture NurOwn for patients with amyotrophic lateral sclerosis (ALS). For over 85 years, Catalent…
On October 6, 2020, molecular engineering company Scribe Therapeutics announced an exciting partnership. Originally founded by molecular engineers and CRISPR inventors, Scribe Therapeutics seeks to develop in vivo genetic medicine to treat…
According to a press release, biopharmaceutical company Apellis Pharmaceuticals ("Apellis") recently launched registration programs to explore pegcetacoplan for patients with C3 glomerulopathy (C3G), amyotrophic lateral sclerosis (ALS), and immune…
As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…
According to a story from BioSpace, the biopharmaceutical company Clene Nanomedicine, Inc., has recently announced that it has completed the process of enrollment patient participants in its upcoming phase 2…
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