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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…
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…
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…
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…
The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…
According to a story from CNBC, the now former Commissioner of the US Food and Drug Administration (FDA) Scott Gottlieb was recently featured on the news network for a discussion…
LLUCH Loma Linda University Children's Hospital (LLUCH) is one of six hospitals in the University's health system. They are a faith-based system located in Southern California. They are the only…
The FDA previously announced their plan to hire 50 new employees specifically to manage the influx of gene therapy applications. Currently, they have approximately 800 applications awaiting review and they…
Sickle cell disease (SCD) is a rare blood condition. It causes sickling in the red blood cells which results in the blockage of blood vessels and capillaries. The sickling process…
CRISPR Therapeutics and Vertex Pharmaceuticals have been collaborating to develop a new treatment option for Sickle Cell Disease. It's called CTX001, a type of stem cell therapy. This drug has…
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…
Sickle cell disease (SCD) is a blood disorder. SCD patients have abnormal hemoglobin, or sickle hemoglobin, caused by a genetic mutation in the beta-chain. It results in deformed and inflexible…
Rare disease research is often underrepresented in the field of science. However, recent developments have made researchers more interested in studying this group of diseases. For instance, investments in gene…
The United States Food and Drug Administration has accepted an Investigational New Drug application (IND) for an investigational gene therapy designed to be a treatment for sickle cell disease. Previously,…
Ebony.com featured a story this September of a young middle school girl named Kennedy Cooper who has sickle cell disease (SCD). Sickle cell diseases are inherited disorders that affect the…
The Noah’s Ark Foundation For Sickle Cell, an NGO based in Lagos, Nigeria, is working to support people with sickle cell disease. In a recent interview with the Nigerian Tribune,…
September was Childhood Cancer Awareness Month and Sickle Cell Awareness Month. To mark to occasion, the Aflac Cancer and Blood Disorders Center at the Children’s Healthcare of Atlanta released a…
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