09.30.2024 BridgeBio Pharma Surpasses Interim Analysis Enrollment Target and Receives U.S. FDA Rare Pediatric Disease Designation for BBP-418, a Potential Treatment for Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)07.18.2024 SEE MORE ...
Improved understanding of tumor biology through molecular alteration and genetic advances has resulted in a number of major changes in the 2020 World Health Organization’s (WHO) classification of bone tumors. These changes include the reclassification of the existing tumors and the introduction of seve...
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This step is being taken out of an abundance of caution after six women in the United States between the ages of 18 and 48 experienced severe blood clots that occurred between 6 to 13 days after vaccination. These instances appear to be extremely rare—the six reported cases were among the...
Eosinophilic esophagitis is a poorly chosen term used to describe a form of hypersensitivity limited to the esophagus. It is increasingly recognized among pediatric patients and adults, particularly young men. Patients may have symptoms that simulate gastroesophageal reflux disease, although many present ...
a spectrum of participants were included, some with the disease or condition of interest and some without objective diagnoses were made using the "gold" standard or current clinical standard for diagnosis of the disease or condition participants received both the new test and some form of the diag...
ADA-SCID is a very rare and life-threatening disease caused by the alteration of a single gene and affects approximately 350 children worldwide. MolMed, GSK team up on development of production process for gene therapy More results ► Acronyms browser ? ▲ AD/PL AD/PVAD AD/SDAT AD/T AD...
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Your life is organized like an orbit. I know. 了解了这点,就知道为何四年于相隔千里的我们,似乎只是弹指一挥间而已。 我其实了解小女儿的心理,是希望多一点surprise多一点romantic多一点傻里吧唧的指天划地的。可惜我这方面的才能似乎多用在0,1组成的无机世界里了,于是只能委屈她了。大多数的时间,她就一...
As I sit at my desk, I begin to wonder: what is gene therapy? What makes it so different from current medications? What types of disease states are being targeted by gene therapy and, finally, if these medications are truly cost effective at a price point of $4 million, how will the...