Clinical studies are critical tools in identifying and developing therapeutics for a wide variety of conditions. This research is especially important in the rare disease and chronic illness spaces,…
The goal of providing precision techniques to cancer care is becoming increasingly complicated. According to a recent article in Inside Precision Medicine, the cause may be attributed to the…
Historically, drug development within the rare disease space has been stifled; companies tend to focus on developing therapies for bigger patient populations that will be more impactful and more…
CRISPR was first described ten years ago as a viral mechanism for defense, taken from bacteria, which could be transformed into genetic scissors and accurately alter DNA. About Casgevy CRISPR…
According to a story from Pharmaceutical Business Review, the UK's National Institute for Health and Care Excellence (NICE) is backing daridorexant (marketed as QUVIVIQ) as a treatment for chronic insomnia…
Patients with severe asthma with an eosinophilic phenotype who are 12 years of age or older may now receive treatment with FASENRA as an add-on maintenance treatment. Note that…
J.D. Martinez is a designated hitter and outfielder for the LA Dodgers. According to a story from The Santa Clarita Valley Signal, he also lives with asthma, which can have…
Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…
In 2005, the U.S. Food and Drug Administration (FDA) approved abatacept for the treatment of moderate-to-severe rheumatoid arthritis. Twelve years later, in 2017, the FDA expanded the drug's approval…
Arcus Biosciences and Gilead Sciences issued a press release announcing encouraging overall response rates and six-month progression-free survival rate for their combination treatment of domvanalimab plus zimberelimab and chemotherapy…
Cancer immunotherapy has transformed the landscape of cancer treatment, revealing that not all patients with solid tumors respond effectively to this approach. As reported by News Medical Life Sciences,…
In the past, the U.S. Food and Drug Administration (FDA) approved Opdivo (nivolumab) for different forms of cancer, such as non-small cell lung cancer (NSCLC) and melanoma. More recently,…
Professionals from all corners of the world convene at SSIEM each year to discuss the latest groundbreaking discoveries. According to a report in BioSpace, one such breakthrough was presented by…
The American Society for Bone and Mineral Research (ASBMR) held its Annual Meeting from October 13 to 16, 2023. During the meeting, over 2,000 attendees and stakeholders came together…
Right now, there are no disease-specific treatments or cures for Angelman syndrome, a rare neurogenetic syndrome. Research centers around exploring options to reduce seizures, improve sleep, and contribute to…
If Wave Life Sciences’ recently submitted trial application for WVE-006 is approved, it will become the first RNA editing treatment in a clinical development setting. Wave’s CEO, Paul Bolno,…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
The American Society for Bone and Mineral Research (ASBMR) and Rare Bone Disease Alliance (RBDA) held the annual Symposium on Rare Bone Diseases this year on October 12. During…
Aminosalicylates are a type of drug often used to manage inflammatory bowel diseases (IBD) like ulcerative colitis (UC). The Crohn's & Colitis Foundation explains that these therapies, which contain…
The xenotransplant involved transplanting Yucatan miniature pig kidneys into macaque monkeys with 69 genetic modifications. One monkey survived for over two years, offering hope to the team of Harvard…
Currently, there are no FDA-approved treatments designed for cognitive impairment related to Huntington’s disease. However, to improve the lives of those affected, additional research and therapeutic development are urgently…
Urinary tract infections, or UTIs, are infections that affect the urinary system, including the urethra, kidneys, and bladder. The majority of UTIs are caused by E. coli bacteria (or other…
The two trials evaluating lacutamab, an investigational therapy developed to treat T-cell lymphomas, were put on partial hold by the FDA. The patient died of hemophagocytic lymphohistiocytosis, which is…
According to a story from Medical Xpress, a recent study found that pelvic floor muscle training provided no additional advantage over bladder training in women with symptoms of overactive bladder.…
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