As recently reported in CheckOrphan, a new drug for sickle cell disease has quickly ascended through the FDA approval processes in an accelerated review. A New Sickle Cell Drug Shrouded…
According to a story from PMLive, the US Food and Drug Administration (FDA) has recently begun the priority review process for a new potential therapy from pharmaceutical behemoth Novartis. This…
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Calls to Ban CRISPR A scientist named He Jiankui recently shocked the scientific world with his announcement that he had created twin “CRISPR babies”. The babies, a result of…
According to a story from BioSpace, the biopharmaceutical company Imara, Inc. has recently announced that its experimental product candidate IMR-687 has earned Fast Track designation from the US Food and…
According to a story from MedCity News, a patient with the rare blood disorder beta thalassemia will be the first patient dosed in a Phase I/II clinical trial involving a…
According to a story from Nature, Dr. Elliot Vichinsky thinks that almost a third of his patients with sickle cell disease ultimately die from causes that could be prevented. Sickle…
A diagnosis means everything for a rare disease patient. However, to process 200 patient records manually, it would take the most dedicated physician approximately 40 hours. That's one full work…
One Chinese scientist has completed a research project that has shocked many in the medical community. Two twin baby girls born in November of 2018 where the recipients of his…
This past Sunday, December 2nd, during the Patriot's game against the Minnesota Vikings, 20 players wore customized cleats to spread awareness for various philanthropic causes. This is the third year…
According to a story from Business Wire, the drug developer Bioverativ, Inc., recently announced its intention to present data related to its latest developments in blood disorder therapies. This data…
According to a story from Greenville Online, the Greenville Health System has begun implementing a comprehensive treatment program for patients with sickle cell disease and its most severe and common…
According to a story from EurekAlert!, a collaborative team of researchers from the University of Florida and The Salk Institute are using cryo-electron microscopy in order to more closely examine…
According to a story from MPR News, Rae Baylark, who is the founder and current president of the Sickle Cell Foundation of Minnesota, felt like she had nowhere to seek…
The Young family from Oregon, U.S. have used genetic screening to test their embryos for a variation of the BRCA gene that significantly increases the risk of breast cancer, reports…
The hemoglobin A1C test is a test that is commonly used to determine if patients have diabetes. A study last year in February 2017, by the American Medical Association found…
In Cincinnati, there are two children, Taryn and King Walker. They are 13 and 11 years old respectively. Taryn and King are excellent students and attend the School for Creative…
Patients with sickle cell disease often face stigma about their need for pain medication because care providers are suspicious of drug abuse, reports The Dallas Post Tribune. Although this is…
Now these are the kind of stories we love to read about! Canadian Revée Agyepong is the first adult in Canada to be cured of sickle cell anemia through stem cell…
According to a story from bioportfolio.com, The California Institute for Regenerative Medicine has given $5.74 million to the City of Hope, a private, nonprofit clinical research center that is primarily…
People living with sickle cell disease should have a new option for treatment in the not-too-distant future. In addition to the blood transfusions and bone marrow transplants from healthy donors, a…
On January 30th, pop singer Jordin Sparks' step-sister Bryanna Jackson-Frias lost her battle with sickle cell anemia, reported People. Complications surfaced that sent her to the ICU. Jordin posted to…
A couple of days before Christmas, the FDA granted a regular approval to the drug, hydroxyurea (Siklos) for treatment of pediatric sickle cell anemia. Sickle cell anemia is a chronic…
Morning, noon, and night Nikki Peterson experiences varying levels of pain, according to an online article in The Root. The pain affects her mood and how she approaches every day.…
Some breathtaking things are happening in the world of sickle cell anemia (SCA) that could potentially impact the lives of nearly 275,000 babies around the world and nearly 100,000 African…
Sickle cell anemia results from a single mutation in the hemoglobin gene. The mutation creates a protein which has an atypical structure. This altered structure throws a wrench in the…
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