According to a story from Psychology Today, one of the most unique genetic disorders known to science it called Williams syndrome. While the disorder causes intellectual disability and other chronic…
Gaucher Disease Awareness Month takes place every October, but amidst this new global pandemic, planning has taken place early to figure out how to make the month as successful as…
Chemotherapy-induced peripheral neuropathy, or CIPN, is a common side effect of chemotherapy. A recent study aimed to uncover whether or not oxaliplatin chemotherapy increased risk of CIPN for those with…
By Caitlin Seida from In The Cloud Copy CRISPR-Cas9, often called just CRISPR, is a technique used to quickly edit DNA to alter gene functions. Think of it like the…
Janssen Pharmaceuticals has an ongoing Phase 1/2 clinical trial for the inherited retinal disease X-linked retinitis pigmentosa (XLRP). They are investigating an adeno-associated gene therapy called RPGR as a therapeutic…
Scares That Care Scares That Care is a 501(c)(3) organization, first established in 2007 by Joe Ripple, dedicated to fighting the "monsters" of childhood illnesses, breast cancer, and burns. They support…
The EveryLife Foundation has initiated a scholarship fund to help those with rare conditions pursue their academic dreams. These will be one-time awards of 5,000 dollars. Up to 32 patients will…
In a recent webinar hosted by the International Waldenström's Macroglobulinemia Foundation (IWMF) and held on July 22nd, 2020, Dr. Jeffery Matous from the Colorado Blood Cancer Institute gave an in-depth…
BioMarin has recently submitted a Marketing Authorization Application to the European Medicines Agency for their achondroplasia treatment, vosoritide. As of now, the review of the application will begin in August.…
According to a story from Charcot-Marie-Tooth News, a new nonprofit organization called Shark Tooth Cycling has recently begun operations. The mission of the organization is to encourage young people in…
During EHA25 Virtual, researchers presented on hematological concepts and the future of diagnosis, treatment, and research. One such presentation discussed results from the InHIBIT-Bleed study, which examined the efficacy…
Thanks to COVID-19, many annual meetings and other events have been held online this year. One such event was the American Society of Retina Specialists (ASRS) Virtual Annual Meeting,…
When Emilia Galarneau grows up, she'll have quite an exciting story to tell about her birth. According to the Connecticut Children's Medical Center, Emilia was born on March 6th,…
When the team at Freiburg University discovered the domain of the T-cell receptor they were surprised that the newly-coined “RK motif” had never been identified. According to a recent…
According to a story from Globe Newswire, the biopharmaceutical company Ovid Therapeutics and the University of Connecticut School of Medicine (UConn) have announced a new license agreement and research collaboration…
According to a story from The Island Packet, fourteen year old Presley Miller, of Hilton Head Island, SC, was recently confirmed to have died from complications related to the rare…
According to a story from The Philadelphia Inquirer, 25 year old Gabby McNary isn't taking any risks when it comes to the coronavirus/COVID-19 pandemic. Philadelphia was hit hard by the…
According to a story from waff.com, Juliana Goode was first diagnosed with Cushing's disease in September 2018. It was an unusual case as Juliana is a teenager and the illness…
According to a story from gopresstimes.com, Lily Laurent, a 13 year old student at De Pere middle school in Wisconsin, has lived with juvenile idiopathic arthritis for nine years. While…
According to a recent article in the Myasthenia Gravis News, a team of doctors in Japan had been treating a man now aged sixty for multiple myeloma (MM). The…
According to a story from ahusnews.com, traveling with atypical hemolytic uremic syndrome (aHUS) takes some extra preparation and planning ahead. This syndrome can affect multiple organ systems and especially the…
According to a story from Myasthenia Gravis News, a recent case report describes a unique case of a 60 year old male multiple myeloma patient. After receiving treatment for the…
According to a story from Markets Insider, the biotechnology company NMD Pharma A/S has recently announced that the Dutch ethics committee has approved the company's plan for a combined phase…
Lupus nephritis causes kidney inflammation and can often lead to chronic kidney disease (CKD). An issue that medical professionals have faced is predicting the likelihood of individual patients developing CKD.…
According to a story from msn.com, two women who were impacted by the rare disease familial chylomicronemia syndrome (FCS) have lead the charge in changing the procedures used by the…
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