According to a story from interestingengineering.com, the capabilities of CRIPSR gene editing technology have been creating a buzz in the medical field for years now, with many seeing the technology…
Unfortunately, many people with sickle cell disease are at an increased risk of experiencing central nervous system complications that can reduce quality of life. But more importantly, complications like stroke,…
As COVID-19 continues to spread across the globe, more and more people are wondering how this virus might affect them. This is especially true for patients with rare diseases,…
As reported in Biospace, Fulcrum Therapeutics has announced it will be submitting an application to the FDA for FTX-6058 as an Investigational New Drug (IND) by the end of 2020…
Fulcrum Therapeutics has recently announced that it is on track to submit an Investigational New Drug application for their medication, FTX-6058. The application will likely be filed in the second…
In a post from MD Magazine, a video from the HCPLive Network depicts a conversation between a number of sickle cell anemia experts about different clinical presentations and complications associated…
Sickle Cell Disease Sickle cell disease is a rare blood disorder. It is progressive, debilitating, and sadly can be life-threatening. The genetic defect leads patients' red blood cells to be…
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An article published earlier this year by Fierce Biotech outlines an approach that was developed by researchers at the Fred Hutchinson Cancer Research Center. Using CRISPR-Cas9’s gene-editing method the researchers were…
No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work,…
Copyscape score: 9% Deondra Clark was unable to have many of the experiences that other children had. Homecoming, prom, sports, and other childhood activities were not a possibility for her,…
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At times there seems to be no happy medium in life. A recent article in Bloomberg claims that the speed at which the FDA is approving newly developed drugs is…
There is no known cure for sickle cell disease. Treatment is symptomatic and meant to prolong life. While a blood and bone marrow transplant is an option, not everyone…
Grajevis Bakatunkanda lived in the Democratic Republic of the Congo, where he received treatment for malaria after experiencing weekly pain crisis. The treatment he received did not work, as…
As originally reported in Medical Xpress, CRISPR has been a hot conversation starter in recent years. It has been framed as gene editing made easy: cheaper and simpler than previous…
CRISPR/Cas9 technology reached another milestone with encouraging results in two recent trials of CTX001. An article in Pharmaceutical Technology carried an announcement by CRISPR Therapeutics and Vertex Pharmaceuticals about…
Global Blood Therapeutics was recently granted priority review by the FDA for their new drug Oxbryta. This drug is meant to treat sickle cell disease, and it is the…
According to a story from The Oklahoman, Dr. Roger McEver, who is a scientist and the Vice President of Research at the Oklahoma Medical Research Foundation (OMRF), played a pioneering…
New knowledge of the genes that affect sickle cell disease and beta thalassemia has allowed for advances in treatments for these diseases. Advances in technology have also helped with…
For people with sickle cell disease, a common, painful symptom is vaso-occlusive crisis, which is when blood circulation is blocked by sickled red blood cells. This restriction of circulation results…
The Immunization Action Coalition (IAC) Express Newsletter reports that 981 cases of measles have been reported so far this year, with 41 new cases added just last week. Rare disease kids…
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…
It is 2019, and minority populations still account for fewer than 10% of clinical trial participants. Two decades ago, the "NIH Policy and Guidelines on the Inclusion of Women and…
Hereditary Hemochromatosis Hereditary hemochromatosis (HH) is a rare disease caused by hepcidin deficiency or hepcidin insensitivity. Hepcidin naturally regulates iron absorption/distribution in the body. Without hepcidin, HH patients suffer from…
The Foundation for Sickle Cell Disease Research's 13th Annual Research & Educational Symposium Fort Lauderdale, FL Are you a sickle cell disease patient? Well don’t miss this important patient event.…
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