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.…
AstraZeneca is ending a phase 3 trial of Ultomiris, which was being evaluated as a treatment for amyotrophic lateral sclerosis (ALS). The pharmaceutical company acquired the C5 inhibitor after buying…
In 1970, ketamine was used to treat injured soldiers on the tail end of the Vietnam War. The treatment helps relieve pain, but doesn't affect cardiac or respiratory function.…
Lisa Stockmann Mauriello began her petition to Biogen almost one year ago. In her letter to the developers, Biogen and Ionis Pharmaceuticals, Lisa explained that she has an inherited form…
Following several Patient Worthy articles on the subject earlier this year, Fierce Biotech recently published news that Biogen is preparing to release the Phase III VALOR trial drug, tofersen,…
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…
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