Home2020May 2020 -

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While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar hosted by the events organizer Terrapinn, producer of the annual World Orphan Drug Congress. Denise Cloutier, lab manager at the University of Florida’s Powell Gene...

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A smartphone app that measures hemoglobin levels using pictures of a person’s inner eyelid may help assess anemia in people with blood disorders such as sickle cell disease, a new study reports. The study, “mHealth spectroscopy of blood hemoglobin with spectral super-resolution,” was published in the journal Optica. Hemoglobin is the protein that carries oxygen throughout the body in red blood cells. Laboratory tests of blood hemoglobin levels are done routinely as an initial screening...

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Taking vitamin D supplements may lower the number of visits to the emergency department due to vaso-occlusive pain episodes in African American children with sickle cell disease (SCD), a study found. The findings also showed that 45% of these children with SCD have vitamin D deficiency. The study, “Vitamin D supplementation and pain-related emergency department visits in children with sickle cell disease,” was published in the journal Complementary Therapies in Medicine. Vaso-occlusive pain episodes are the most common reason for...

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European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred in Stockholm, but instead took place online via Zoom. Rare disease patient advocates discuss the benefits of newborn screening at a recent conference. (Photos by Larry...

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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...