News

May 28, 2020May 28, 2020

Smartphone App May Help Assess Anemia Using Eyelid Pictures

News

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 ... Read more

May 26, 2020May 26, 2020

Vitamin D Supplements May Reduce Pain-related Emergency Room Visits in Children with SCD

News

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 ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

News, syndicated

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 ... Read more

May 22, 2020May 22, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

cystic fibrosis, News, syndicated

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 ... Read more

May 21, 2020May 21, 2020

Partnership to Ensure Supply of LentiGlobin, Potential Gene Therapy

News

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 ... Read more

May 19, 2020May 19, 2020

Sickle Cell Disease Association, Hemanext Join to Support Programs

News

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 ... Read more

May 18, 2020May 18, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News, syndicated

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. ... Read more

May 14, 2020May 14, 2020

Agios to Decide Next Steps for Mitapivat as SCD Treatment by Mid-year

News

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 ... Read more

May 13, 2020May 13, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

News, syndicated

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. ... Read more

May 12, 2020May 12, 2020

Gene-editing Therapy CTX001 Granted RMAT Status by FDA for Severe SCD

News

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 ... Read more