A recent release published in BioSpace announced that a gene therapy company is vigorously pursuing the study of AVB-101 to treat patients with frontotemporal dementia (FTD), including FTD with GRN…
The increasing penetration of digital technologies and the ubiquity of smartphones and the internet have transformed several spheres of our lives. Healthcare is no exception. With technological advances, it has…
In 2013, the Morells welcomed two identical baby girls, Kaylie and Kenzie. The joy they felt soon disappeared as the twins began to slip back from their normal infant and…
Bladder cancer patients and their families have faced years, and even decades, of waiting for a breakthrough in bladder cancer treatment. For some patients, the results of two clinical…
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disorder in which blood cells break apart prematurely. In an effort to bring more effective and targeted treatments…
Neuroblastoma most commonly occurs in children under the age of five. The disease usually affects areas of the adrenal gland (positioned over the kidneys) or areas in the neck, chest,…
Roche has added another failure this year with its drug gantenerumab. As noted in Fierce Biotech, the drug was unable to improve functional or cognitive decline in two Phase…
The biotechnology company Cabaletta Bio has recently announced that the company investigational new drug (IND) application for its experimental CD19-CAR T-cell therapy CABA-201 has been cleared by the US Food…
Prior to the FDA’s approval this month, nirogacestat (Ogsiveo) received the following FDA designations applicable to the treatment of desmoid tumors: Breakthrough therapy providing a rapid review. Fast track to…
According to a story from The Print, Neha Shoriwala, a mother from New Delhi, is urgently seeking help for her son Harshil, who lives with Wilson's disease, a rare illness…
When drugs enter into clinical studies, both patients and drug developers alike hope for success. These therapies could transform the treatment sphere, especially in disease states with limited or…
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…
The FDA has granted IDEAYA Biosciences fast track status for IDE161 as a treatment for HR+, Her2- breast cancer. The therapy is a selective inhibitor of ADP ribose glycohydrolase…
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…
Every three to four minutes, someone in the U.S. is diagnosed with a blood disease such as lymphoma, leukemia, or sickle cell anemia. As reported in the Pittsburgh Post-Gazette,…
Chip Wilson, billionaire founder of Lululemon Athletica, was first diagnosed with muscular dystrophy in 1987 at the age of 32. Wilson, who is now 66 years old told Global…
In 2010, Eddie Vedder (well known for his musical career in Pearl Jam) co-founded the EB Research Partnership (EBRP) alongside wife Jill and a group of parents whose children…
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…
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,…
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…
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