What causes developmental disorders? How can the human genome play a role in diagnosing and treating these conditions? Well, a team of scientists from the Salk Institute recently set…
According to a story from mlive.com, the state of Michigan has confirmed its first case of acute flaccid myelitis, a rare disease that tends to affect children, this year. There…
INO-3107, a treatment for recurrent respiratory papillomatosis (RRP), has recently received the Orphan Drug Designation from the FDA. This treatment is currently being evaluate in a Phase 1/2 trial by…
Rare Pediatric Disease Designation has just been granted by the FDA for an investigative therapy for lymphatic malformations (LMs). This therapy is called TARA-002 and was developed by Protara Therapeutics.…
An investigative therapy for hemolytic disease of the fetus and newborn (HDFN) has just received the FDA's Rare Pediatric Disease and Orphan Drug Designations. This therapy is called nipocalimab and…
In a recent webinar hosted by the National Organization for Rare Disorders (NORD), a number of speakers discussed an up and coming project spearheaded by the organization: the Rare Disease…
By Danielle Bradshaw from In The Cloud Copy Shimmer Research has announced that OWEAR - the Open Wearables Initiative - has decided to make their health technologies, like their database…
Moebius Syndrome Moebius syndrome is a rare condition that causes an inability to move the eyes laterally and facial paralysis. A similar type of paralysis can also occur as a…
Fast Track Designation and Rare Pediatric Disease Designation have just been awarded from the FDA to HemoShear Therapeutics. This designation was given to their new investigative therapy for propionic acidemia…
By Lauren Thayer from In The Cloud Copy COVID-19 has brought life to a screeching halt for everyone across the world. Schools cancelled, work transitioned to remote, all activities cancelled,…
According to a story from PR Newswire, the biotechnology company INOVIO has just announced that the company's investigational product candidate INO-3107 has earned Orphan Drug designation from the US Food…
According to GlobeNewswire, BridgeBio Pharmaceuticals and the Ivy Brain Tumor Center have recently began their Phase 0/2 trial of infigratinib. This treatment is meant for those with recurrent glioblastoma that…
By Lauren Thayer from In The Cloud Copy Systemic mastocytosis (SM) is a complicated condition of the blood in which excessive numbers of mast cells (a type of white blood…
Just last month, the FDA approved MYCAPSSA for patients with acromegaly who have previously been treated with octreotide or lanreotide. As the first FDA-approved oral somatostatin analog, developer Chiasma…
By Danielle Bradshaw from In The Cloud Copy The chances of your child having Jordan’s syndrome are incredibly small. That, however, was not the reality for Joe Lang and his…
According to a story from the Columbus Dispatch, Brian McNeal has been living with trigeminal neuralgia for four years. The rare disease leaves him in constant facial pain. The chronic…
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.…
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