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Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, Maximilian, has cystic fibrosis (CF), making him particularly susceptible to COVID-19 and the complications that can arise from it. As he complies with stay-at-home orders and remains in social isolation during the pandemic, video games have helped him stay connected with the outside world from...

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Living with sickle cell disease, I rely on several medications and treatments for various situations. In general, I take medications for my own well-being, as research shows that sickle cell patients who struggle to comply with medication regimens tend to experience a lower quality of life. Ergo, to have the best quality of life I can despite this debilitating disease, I must be compliant with my medication regimen. My medication is essentially split into three...

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Bluebird Bio extended a partnership with two divisions of Hitachi Chemical for the long-term development and manufacturing of LentiGlobin, its investigational gene therapy for sickle cell disease (SCD), and other potential and approved treatments. The two subsidiaries — Hitachi Chemical Advanced Therapeutics Solutions (HCATS) and Apceth Biopharma — will be in charge of late-clinical development and manufacturing of LentiGlobin at facilities in Germany (Apceth) and the U.S. (HCATS). “It is our honor to support bluebird bio in the manufacture of their potentially transformative...

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My parents knew they were carriers of the sickle cell trait before I was born, so they made an effort to find out if I had the disease during the prenatal stages of my mother’s pregnancy. Once they found out I had it, plans and preparations were made to ensure they knew how to be effective caregivers from the moment I was born. My parents certainly tried their best to make sure they kept me...

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Hemanext, a medical technology company, is working with the Sickle Cell Disease Association of America (SCDAA) to help people with sickle cell disease through a mix of educational programs, grassroots events, and public-awareness campaigns. “During these very difficult times in the midst of the COVID-19 crisis, we appreciate and look forward to partnering with Hemanext to improve the lives of people with sickle cell disease and their families,” Beverley Francis-Gibson, SCDAA’s President and CEO said in a press release. ...

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Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in Stockholm, instead took place via Zoom. Eurordis and its co-organizer, Orphanet, used the occasion to appeal to the European Union in Brussels to urgently approve standardized policies...

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Agios Pharmaceuticals announced that, despite the current COVID-19 outbreak, it remains on track to decide by mid-year next steps for mitapivat (AG-348) as a potential treatment of sickle cell disease. Mitapivat is in an ongoing proof-of-concept Phase 2 trial in up to 25 patients with stable SCD, but further enrollment has been suspended due to the pandemic. “We moved quickly to reduce the risk of our team’s and communities’ exposure to the virus and took action to enable uninterrupted access to...

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The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other disease-related issues and those brought by the pandemic. Developed and led by the RDCRN Data Management and Coordinating Center at Cincinnati Children’s Hospital Medical Center, the research survey...

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One of the most widely discussed symptoms of sickle cell anemia is a sickle cell crisis. This is an episode of pain caused when sickle-shaped red blood cells block the body’s blood vessels and prevent oxygen from flowing. One can read the biological explanations of such a crisis, but can’t really understand what it feels like without having the disease. So, it is only natural for people to wonder about it. I’m constantly asked what...

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The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to CTX001, an investigational gene-editing cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are working together to develop CTX001. RMAT is given to expedite the development and review of new therapies whose main purpose is to treat medical conditions considered serious or...

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