A study recently published in Cell Stem Cell has shown that using human stem cells in combination with medical chemistry can improve existing medications to treat genetic disorders. Dr. Alfred George Jr.…
According to a story from BioSpace, the biotechnology company Ascentage Pharma announced that two of its investigational treatments have recently received Orphan Drug designation from the US Food and Drug…
by Lauren Taylor from In The Cloud Copy A comorbidity occurs when there is a presence of one or more additional illnesses or conditions that co-occur with the primary condition.…
Later this week, from October 21-23, presenters and attendees will be present at the 34th Annual North American Cystic Fibrosis Conference (NACFC). Although this year is slightly different, considering the…
by Lauren Taylor from In The Cloud Copy Latent autoimmune diabetes in adults (LADA) is a less often talked about form of diabetes that occurs as the result of the…
According to a story from BioSpace, a recent study conducted by the biotechnology company Goldfinch Bio in collaboration with academic partners is highlighting the importance of proteinuria in determining outcomes…
On October 15th, 2020, the Rare Disease Legislative Advocates (RDLA) held a webinar discussing some of the latest news regarding ongoing legislation that is most relevant to the rare disease…
by Lauren Taylor from In The Cloud Copy The human papillomavirus (HPV) vaccine has been being administered to teens and young adults worldwide for over a decade now. It has…
Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…
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