More than two months after Blueprint Medicines Corp. gained a broadened label for Ayvakit (avapritinib), the company is “really pleased to see the diversity of revenue this early in the launch across specialties and across both the academic and community settings,” said Philina Lee, chief commercial officer. The situation “bodes very well for a continued cadence of prescribing [into] the foreseeable future,” she said.
Non-profit Solve GNE LLC has raised over $2.5 million and announced sponsored research agreements to help advance research in hereditary inclusion body myopathy (HIBM), or GNE myopathy (GNEM).
Positive top-line phase II results caused Mymd Pharmaceuticals Inc.’s stock (NASDQ:MYMD) to surge 31.8% on July 31 to close at $1.45 per share. The data behind the push were for MYMD-1 (isomyosmine) for treating chronic inflammation associated with sarcopenia.
Sarcopenia is the loss of muscle mass typically in the elderly that ultimately reduces mobility, diminishes quality of life, and can cause potential fall-related injuries that can become serious. While exercise is an appropriate approach, unfortunately patients with underlying health conditions and low motivation are unlikely to adhere to regimented muscle training. Moreover, there are no available effective cures for addressing age-related muscle loss. Still, it is possible that a failure to repair muscle after injury may be a major contributor to muscle mass loss in the aged that can be addressed by using muscle stem cells.
Epibone Inc. has received IND clearance from the FDA to begin testing its engineered allogenic osteochondral graft in humans. Clinical trials are set to open patient recruitment as soon as early next year.
Tissue regeneration company Osteopore Ltd. is on the cusp of dramatically changing the way orthopedic surgery is conducted globally, with Australia playing a crucial role. Osteopore was the first company to successfully develop and commercialize 3D-printed bioresorbable implants for surgical use, reducing post-surgery complications compared to permanent implants.
A new study on the interleukin 1 (IL-1) pathway and its involvement in systemic inflammation and autoinflammatory diseases has described a mutation of the IL-1 receptor type 1 (IL-1R1) receptor associated with chronic recurrent multifocal osteomyelitis (CRMO). The variant p.Lys131Glu of this receptor was identified in a patient with CRMO. A peripheral blood mononuclear cell analysis showed an inflammatory event in lymphocytes, especially in monocytes and neutrophils. The research was conducted by a group of scientists at Zhejiang University in China and reported in the July 11, 2023, issue of Immunity.
Beigene Co. Ltd. has identified 3, 4-dihydroisoquinolin-1 (2h)-ones derivatives acting as stimulator of interferon genes protein (STING; TMEM173) antagonists reported to be useful for the treatment of systemic lupus erythematosus (SLE).
Researchers from Ermium Therapeutics SAS have presented preclinical data for the novel chemokine CXCR4 receptor ligand ER-000145, being developed for the treatment of type 1 interferonopathies, such as systemic lupus erythematosus (SLE).
Researchers from the Institute of Translational Genomics at Helmholtz Munich have described a genetic overlap between type 2 diabetes (T2D), a disease that is also associated with obesity, and osteoarthritis, a degeneration of the joints that worsens with age and coincides in the factor risk of being overweight. The researchers used genetic data, multiomics and functional analysis of the tissues T2D and osteoarthritis express to identify which genes were associated and correlated with both diseases. They published their results on July 10, 2023, in The American Journal of Human Genetics.
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