Amyotrophic lateral sclerosis (ALS) is a rare progressive motor neuron disease that, due to its high complexity, still lacks effective treatments. Development of a new drug is a highly costly and time-consuming process, and the repositioning of approved drugs can represent an eff...
We have included all novel drugs approved by these three agencies up to December 31st, 2023. We also accessed “The IUPHAR/BPS Guide to PHARMACOLOGY” (https://www.guidetopharmacology.org/), an informative go-to site to clarify the pharmacological mechanism of action of molecules based on ind...
Through research in mice, scientists have found that proteins at the blood-brain barrier pump out riluzole, the only FDA-approved drug for ALS, or Lou Gehrig's disease, limiting the drug's effectiveness. However, when the investigators blocked these proteins, the effectiveness of riluzole increase...
2023) with one exception. Unlike in previous years, we have included cellular and gene therapies approved by the FDA (U.S. Food and Drug Administration2023a,b) into our analysis. However, we exclude generics, or generic versions of
Dronabinol (Marinol and Syndros) is also approved to treat anorexia (loss of appetite) associated with weight loss in patients with AIDS (Acquired Immune Deficiency Syndrome).Epidiolex Epidiolex (cannabidiol), also called CBD, is a cannabinoid product approved by the FDA in June 2018. It is ...
Qalsody (tofersen) is a prescription medicine used to treat amyotrophic lateral sclerosis (ALS) in adults who have a specific genetic mutation in the superoxide dismutase 1 (SOD1) gene. Qalsody is the first FDA-approved treatment that targets a genetic cause of ALS to slow progression of ...
This is the only drug approved to treat PPMS. Natalizumab is specific to the α4 integrin subunit on lymphocytes and prevents lymphocytes from entering the CNS, whereas alemtuzumab selectively binds CD52 antigen on lymphocytes, leading to their depletion [129]. Besides those mAbs, sphingosine 1-...
Dramatic stories about weight loss seem to be everywhere on social media. Jennifer Huber, who shared her own story online, has lost more than 50 pounds in five months after starting Mounjaro, an injectable drug approved to treat her Type 2 diabetes. "It's this miracle," Huber said. "I...
Riluzole was approved for the treatment of ALS in 1995. Since the drug is has been already been tested for toxicity and undergone scrutiny by FDA prior to approval – a new application of this drug could move faster in the clinic if future results are encouraging. ...
A screening assay of FDA-approved drugs identified ceftriaxone and other β-lactam antibiotics as potential therapies for amyotrophic lateral sclerosis (ALS) and other neurologic disorders. The results of experiments in a mouse model of ALS provide support for this hypothesis and suggest that ceftriaxon...