The pharmaceutical development company Noxopharm and its majority-owned subsidiary, U.S. biotechnology firm Nyrada, have discovered a potent inhibitor of the protein IRAK4, a “master switch” critical to the development of inflammatory autoimmune diseases, such as amytrophic lateral sclerosis (ALS). IRAK4 has emerged as a key switch in the…
My ALS diagnosis was in 2010. In the months following, I tried to learn as much as possible about the condition and how it would affect my life going forward. I discovered that medical experts knew almost everything about the physical progression of ALS and what to expect…
Specific patterns of a misfolded protein — superoxide dismutase 1 (SOD1) — are found in the brain and spinal cord of patients with sporadic amyotrophic lateral sclerosis (ALS), according to a new study, supporting research suggesting that misfolded SOD1 plays a role in this ALS subtype. The research, “…
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…
Adding Azilect (rasagiline) — a medication already approved to treat Parkinson’s disease — to Rilutek (riluzole) may be able to slow the advancement of fast-progressing amyotrophic lateral sclerosis (ALS), a Phase 2 trial suggests. However, its positive safety results are being challenged by other physicians, who raise concerns that…
Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are caused by different patterns of inflammation in the brain, which may help explain why these two illnesses are so distinct. The German study, “Different neuroinflammatory profile in amyotrophic lateral sclerosis and frontotemporal dementia is linked to the…
My first symptomatic expression of ALS may have been as I left a football game. I fell flat on my face. At the time, I blamed it on the sun and the beer, although that combination in the same dosages had not felled me before. Shortly after, I became prone…
A newly developed method can help characterize the types of misfolded proteins present in patients with amyotrophic lateral sclerosis (ALS), according to findings in a thesis. The recently published doctoral thesis, titled “Structural investigation of SOD1 aggregates in ALS: identification of prion strains using anti-peptide antibodies,” was written…
Sometimes living with ALS makes me feel like I’m competing in the Olympics. I wake up in the morning and wonder how my body will perform that day. Then I lie awake at night rehashing all the things that went wrong. The voices in my head often sound…
Retrotope is providing its investigational fatty acid being developed for Friedreich’s ataxia, RT001, to people with amyotrophic lateral sclerosis (ALS) under an expanded access program, also called “compassionate use.” Individual patients with life-threatening or severely debilitating diseases can petition the U.S. Food and Drug Administration, through their physicians,…
