According to a story from Charcot-Marie-Tooth News, an all new protocol first described in the journal Brain Sciences could mark a significant advance in Charcot-Marie-Tooth disease research. The protocol allows for the…
According to a story from GlobeNewswire, the first patient has been dosed in a recent clinical trial testing the experimental therapy tasquinimod as a treatment for multiple myeloma, a rare…
A recent article published in EMS WORLD cautions that long QT syndrome (LQTS), a potentially lethal disease in young people, often remains undiscovered. An ECG can detect the disease,…
When Zika virus appeared in the news in 2015 and 2016, one of the biggest warnings was for pregnant women: be careful, as this can cause birth defects. Most…
According to a story from BioSpace, the biotechnology company Tyme, Inc. has just announced that it has earned Orphan Drug designation for its experimental product candidate SM-88. SM-88, also known…
August is recognized as Spinal Muscular Atrophy Awareness Month. The goal of this event is to help spread information about this rare disorder among the general public and the broader…
Currently, over 200 different mutations are linked to the development of best vitelliform macular degeneration (BVMD, or Best disease). But while certain treatments may treat certain mutations, there has…
According to a story from GlobeNewswire, the biopharmaceutical company GW Pharmaceuticals plc recently announced that its CBD oral solution Epidiolex® has recently been approved as a treatment for seizures associated…
Last month, at the American Academy of Dermatology (AAD) Virtual Annual Meeting, Dr. Dedee F. Murrell, MD, explained the efficacy of rilzabrutinib for treating pemphigus. According to MDedge, many…
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…
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