According to a story from BioSpace, the biotechnology company Ascentage Pharma has just announced that it has been given Orphan Drug designation from the US Food and Drug Administration (FDA).…
According to a story from theindychannel.com, brothers Elias, age eleven, and Emmett, age seven, were both born with the rare bleeding disorder hemophilia A. However, this has not stopped them…
According to a story from Korea Biomedical Review, Seoul National University Hospital (SNUH) in South Korea has just announced recently that the process of recruiting patients for the National Bio…
According to a story from Buzzfeed News, a recent test of an experimental COVID-19 vaccine being developed by Moderna Therapeutics demonstrated that it was able to produce antibodies in all…
ChemoCentryx has just announced that they have submitted their New Drug Application to the FDA for a novel ANCA-associated vasculitis therapy. It is called avacopan and it's finally something non-steroidal.…
As reported in Parkinson's News Today, three nurses have been awarded $10,000 by The Parkinson’s Foundation to carry out local projects for Parkinson’s patients to better their experience. Each of…
According to a story from PR Newswire, new international treatment recommendation guidelines have recently been updated for nontuberculous mycobacterial (NTM) lung disease. The update pertains to a variant known as Mycobacterium…
According to a story from meaction.net, Dr. Anthony Fauci was recently asked a question about what was being done to address so-called COVID-19 "long haulers," and their potential risk of…
By Danielle Bradshaw from In The Cloud Copy The Lancet Psychiatry has published a review stating that although there have been signs demonstrating that delirium may be a common symptom…
By Lauren Thayer from In The Cloud Copy Fetal alcohol spectrum disorders or FASDs are a group of conditions that can affect a child both physically as well as behaviorally. …
According to a story from keloland.com, Westin Cuka, age six, was first diagnosed with neurofibromatosis, a rare disorder, three years ago. Since then, the family has traveled on multiple occasions…
Biotechnology company Talaris Therapeutics, Inc. submitted an Investigational New Drug (IND) application to the FDA for their novel cell therapy treatment FCR001. As per a press release, the FDA…
Rare pediatric disease designation has just been granted to Rhythm Pharmaceuticals for their investigative therapy called setmelanotide. This drug is being investigated as a therapeutic option for two conditions- leptin…
Each year, June 13th is celebrated as Gastrointestinal Stromal Tumors (GIST) Awareness Day. The ultimate goal of awareness days for rare diseases is to help inform both the general public…
Patients with inoperable or metastasized neuroendocrine tumors (NETs) may now benefit from recently FDA approved peptide receptor radionuclide therapy (PRRT) according to a recent article in MedPage Today. The…
According to a story from thv11.com, Susan Head of Little Rock, Arkansas is a marine veteran who now faces a new battle: Castleman disease. In the midst of the ongoing…
Well-known characteristics of aging are the loss of skeletal muscle mass and a general decline in all of the organs in the body. The speed of decline coincides with the…
After forty years of research to counter or cure acute myeloid leukemia and other blood cancers, a few years ago the medical community exploded with eight new cancer drugs.…
In a recent press release, the U.S. Food and Drug Administration (FDA) announced their approval of Inqovi for patients with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). The…
July is recognized as Juvenile Idiopathic Arthritis Awareness Month. Though arthritis is often considered an illness that is associated with old age, it is estimated that around 300,000 children in…
Launched in January of 2020, and ending in May, the BEAT-PD Dream Challenge was designed to understand whether passive data collected from sensors could track symptom severity and disease progression…
Timber Pharmaceuticals focuses on rare diseases of dermatologic origin. These include scleroderma, congenital ichthyosis, tuberous sclerosis complex, and facial angiofibromas. They are currently working on two clinical trials. Trial 1…
CMT1A Charcot-Marie-Tooth disease (CMT) has a few different subtypes. 60% of all patients diagnosed with type 1 of the rare disease have CMT1A. This subtype means patients have an duplicate copy…
According to a story from The Boston Herald, a new treatment for acromegaly called Mycapssa was recently approved by the US Food and Drug Adminstration (FDA). Developed by Chiasma, a…
A new study published by ACS Nano and conducted by scientists at the University of Illinois at Urbana-Champaign uncovered a new way to modify stem cells. This study was conducted…
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