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…
According to European Pharmaceutical Review, results from a Phase 1/2a clinical trial support the use of AstroRx for patients with amyotrophic lateral sclerosis (ALS). The clinical trial, headed by Kadimastem…
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…
Dilated cardiomyopathy is a heart condition caused by a mutation in the RBM20 gene. This heart condition is unique because it can affect young adults. These individuals are at an…
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…
A series of articles recently published in FierceBiotech describe a discovery by Ohio State and Michigan University scientists. The teams have discovered an immune cell that could be tapped to…
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 story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…
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…
According to a recent article in MedicalXpress, researchers in Japan led by Dr. Shinsuke Ishigaki and his team at Nagoya University, have identified new molecular details about tau’s activity. Alzheimer’s,…
Within the last month, BrainStorm Cell Therapeutics Inc. announced the publication of pre-clinical data on the NurOwn system. NurOwn technology produces autologous, bone marrow-derived mesenchymal stem cells (MSCs) for…
For years, scientists believed there might be a connection between amyotrophic lateral sclerosis and frontotemporal lobar degeneration (FTLD), also sometimes known as frontotemporal dementia. However, researchers from Osaka University may…
Happy Friday! We're about halfway through Stevens-Johnson Syndrome Awareness Month. It's a type of severe skin reaction that often involves admission to a burn unit for treatment. Looking for ways…
Seelos Therapeutics has recently received approval to proceed with phase IIb/III of their trial of trehalose, an investigational ALS treatment. The FDA alerted Seelos of this approval on August 7…
The HEALY ALS Platform Trial, being conducted by Biohaven Pharma, has begun enrolling patients. This study is intended to test the efficacy of three treatments on amyotrophic lateral sclerosis (ALS),…
Scientists and researchers have long known how important glial cells are to the nervous system. Not only do these cells make up a majority of cells within the central nervous…
According to a story from globenewswire.com, the biopharmaceutical company Orphazyme A/S has recently announced that it has submitted its New Drug Application (NDA) for its experimental treatment arimoclomol to the…
CytRx Corporation has recently received the Fast Track designation for their ALS treatment, arimoclomol. This status was also given for the treatment of Niemann-Pick disease and sporadic Inclusion Body Myositis…
What would motivate a man, who has been living with amyotrophic lateral sclerosis (ALS) for twenty years, to accept an invitation to become part of a five-man team and…
Recently, scientists from Emory University set out to discover whether there was any pathological link between spinocerebellar ataxia type 36 (SCA36) and amyotrophic lateral sclerosis and frontotemporal dementia (ALS/FTD). Both…
Biological robots, commonly known as biobots, mimic the actions of other organisms (e.g. jumping or swimming). Biobots are composed of artificial materials and also organic material. As published in…
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.
