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…
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…
Scientists are researching the possibility of fetal gene therapies as possible cures for rare diseases, such as Duchenne muscular dystrophy, hemophilia, sickle cell disease, and Gaucher's disease. With fetal gene…
Researchers have investigated whether self-management electronic health (eHealth) interventions, such as text messaging, apps, and online cognitive-behavioural therapy, should be used to help patients with sickle cell disease self-manage the…
The experimental drug olinciguat (IW-1701) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of sickle cell disease. The full article can…
The experimental gene therapy LentiGlobin for the treatment of severe sickle cell disease is being investigated in an ongoing Phase 1 clinical trial. Bluebird Bio, the company developing the therapy,…
Sickle Cell Disease Research and Educational Symposium "From Biology to Therapy: Improvement of Therapeutic options for clinical care in sickle cell disease" This conference will present the latest research and…
According to Bloomberg, there’s a new big fish in the medical technology pond. Crispr Therapeutics AG jumped in value, multiplied by six, and landed at $3 billion. How is that…
The Investigational New Drug Application for the experimental drug CTX001 for treating sickle cell disease has been put on clinical hold, announced Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics. This hold…
According to a story from apnews.com, sickle cell disease gets very little research or funding despite that fact that about 100,000 people in the U.S. are affected by it. The…
According to a story from Sickle Cell Anemia News, the St. Jude Children's Research Hospital just received $1 million in grant funding from The Links Foundation, which is one of…
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…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
