Digital-native agency closerlook won a bronze Pharma Lion for a docuseries developed with global immunology company argenx that depicts the experiences of people living with myasthenia gravis (MG), a rare and chronic neuromuscular autoimmune disease. The award win for “A Mystery to Me” was announced at the Cannes Lions International Festival of Creativity in France.
Boston-based Alexion Pharmaceuticals announced positive topline data from the company’s Phase III trial of Ultomiris (ravulizumab-cwvz) in adults with generalized myasthenia gravis.
The U.S. Food and Drug Administration placed a clinical hold on Larimar Therapeutics’ lead Friedreich’s ataxia (FA) asset, CTI-1601, after the company reported deaths in the highest dose levels of a nonhuman primate toxicology study.
Biogen and Capsigen forged a strategic collaboration to engineer novel adeno-associated virus (AAV) capsids that have the potential to become transformative gene therapies that treat underlying genetic causes of various central nervous system and neuromuscular disorders.
Option Care Health Inc., the largest U.S. independent provider of home and alternate site infusion services, was selected to participate in the limited distribution network of VILTEPSO (viltolarsen) for patients with Duchenne Muscular Dystrophy who are amenable to exon 53 skipping therapy.
A roundup of some of the latest scientific studies on the novel coronavirus and efforts to find treatments and vaccines for Covid-19, the illness caused by the virus.
Argenx announced positive topline results from the company’s Phase III ADAPT clinical trial of efgartigimod for myasthenia gravis.
Biogen and Sangamo Therapeutics announced a broad global licensing collaboration deal to develop and commercialize several compounds for a range of neurological and neuromuscular diseases.
Roche Holding AG made the company’s second major gene therapy deal in a year, spending $1.15 billion to obtain the rights to Sarepta Therapeutics Inc.’s investigational drug to treat duchenne muscular dystrophy (DMD) outside the United States.
Reata Pharmaceuticals Inc.’s Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met the primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale relative to placebo after 48 weeks of treatment.