News

An experimental oral formulation of edavorone for amyotrophic lateral sclerosis (ALS), called TW001, demonstrated promising pharmacological and safety data in a Phase 1 clinical trial, according to the biotech Treeway. The randomized trial compared TW001 to Radicava (Mitsubishi Tanabe Pharma America), an intravenous medication approved…

AB Science has decided not to seek the re-examination it initially requested after the European Medicines Agency (EMA) issued a negative opinion on marketing authorization for masitinib, the company’s investigational amyotrophic lateral sclerosis (ALS) therapy. The company announced in a press release that the re-examination procedure…

Should scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential rare diseases? Or should researchers restrict their use of genome editing to somatic cells, so that they don’t pass changes on to the next generation? What about stem-cell research, three-parent…

Researchers discovered that a small region of ataxin-2 — a protein known to be involved in amyotrophic lateral sclerosis (ALS) — is crucial for both long-term memory and the formation of toxic aggregates that lead to neurodegeneration. The study with that finding, “RNP-Granule Assembly via Ataxin-2 Disordered…

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

A gene therapy that might treat familial amyotrophic lateral sclerosis (ALS), an inherited form of the disease, was recently granted orphan drug status by the European Medicines Agency (EMA), an award that carries incentives to promote potential treatments for rare diseases. The investigative therapy is in preclinical development…

Two years after approving it, the 28-member European Union will begin enforcing its General Data Protection Regulation (GDPR) — a tough new law that aims to protect the EU’s 512 million citizens, including rare disease patients, from having their medical records misused, sold, or subject to extortion by hackers, third…

Clinigen Group recently partnered with Mitsubishi Tanabe Pharma Corporation to launch an early access program in Europe for Radicava (edaravone), an intravenous treatment for amyotrophic lateral sclerosis (ALS). The therapy is not yet approved in Europe, but the early access program will help make it available…

Voyager Therapeutics presented new data from a study in an animal model of  its clinical candidate VY-SOD101, showing the therapy is effective as it targets the most common cause of familial amyotrophic lateral sclerosis (ALS). The presentation, along with another preclinical program data presentation on a Huntington’s disease therapy…

A new mouse model developed by researchers at Karolinska Institute in Sweden revealed a molecular mechanism that may contribute to the development of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) or multiple sclerosis. The mouse model was described in the study, “Fatal demyelinating disease…