When Rylae-Ann was one year old, her parents had to hold her in an upright position at night in order for her to breathe. Rylae-Ann had a rare genetic disease,…
Neuromyelitis optica spectrum disorder (NMOSD) can have long-term impacts for those affected. Current research suggests that up to 69% of patients may have severe unilateral or bilateral vision loss…
It has historically been difficult to encourage the development of therapies for patients with rare diseases. To overcome this, the Orphan Drug Act was passed in 1983. This act…
Genethon is a non-profit organization committed to the design and development of gene therapies for rare diseases. This unique company is a pioneer in identifying genes relating to genetic…
Have you ever heard of Orphan Drug designation? This designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended to diagnose, prevent, or…
There is no cure for multiple sclerosis (MS). However, treatment advances have helped people to live longer, happier lives. Current therapeutic options include corticosteroids, plasmapheresis, and disease-modifying drugs. Recently,…
In 2020, the U.S. FDA granted Orphan Drug designation to KB407, an investigational gene therapy for patients with cystic fibrosis (CF). More recently, at the start of 2023, the…
From November 10-14, 2022, various stakeholders within the rheumatology sphere connected at the American College of Rheumatology’s (ACR) Convergence 2022. During ACR Convergence, described as the world’s premier rheumatology experience,…
In the European Union (EU), Orphan designation, or Orphan Medicinal Product designation, is granted to drugs or biologics intending to treat, diagnose, or prevent a rare, chronically debilitating, or…
Ten years ago, a biochemist was seeking investors for her new company that was developing mitochondrial-based medicines. The Israeli-born embryologist observed how the blending of mitochondria from one egg…
On January 6, 2023, the FDA granted conditional approval to a new Alzheimer’s drug, lecanemab, that will be sold as Leqembi. Vox carried the original story and included an…
An antidepressant drug that was approved by the FDA in 1959 is being put to new and promising use to fight hard-to-treat breast cancers. The antidepressant, imipramine, was approved to…
Cognitive deficits have been recognized as symptoms of advanced liver disease for many years. As reported in Bioengineer, the American Journal of Pathology recently published a study providing insights…
According to a story from hopkinsmedicine.org, a study evaluating data from 106 patients living with wet age-related macular degeneration found a significant disparity in outcomes when comparing patients that received…
One year ago, in December 2021, clinical studies of lovo-cel for patients who were eighteen or younger were put on partial hold by the FDA. At the time, bluebird…
While alveolar soft part sarcoma (ASPS) is considered to be relatively slow-growing, it is necessary to carefully manage this rare soft tissue sarcoma to avoid metastasizing. Surgery is one…
Continued From Part One Multiple myeloma is caused by the buildup of abnormal white blood cells that form tumors found primarily in bones. Available treatments work for short periods…
Globe Newswire recently published an article announcing positive results from Amolyt Pharma’s Phase IIa proof of concept clinical trial of AZP-3601. Amolyt Pharma, Cambridge, Massachusetts, and Lyon, France, is a…
Part 1 Newswise recently published exciting news of a therapy that causes the immune system to destroy cancer cells in the bone marrow. Researchers at Icahn School’s Tisch Cancer Center…
When the COVID-19 pandemic swept the world, nobody knew what the short- or long-term effects would be. Now, a few years into the pandemic, we’ve begun understanding some of…
According to a story from ca.sports.yahoo.com, the biotechnology company Galecto, Inc. has announced encouraging findings from an intermediate assessment of its phase 2a clinical trial. This trial is evaluating the…
A team of researchers at Shanghai’s Fudan University, acting upon the knowledge that ketogenic diets can boost liver ketone production, have recently examined the possibility of preventing thrombocytopenia (low…
According to a story from empr.com, the Oncologic Drugs Advisory Committee, part of the US Food and Drug Administration (FDA), recently voted 16 to 0 against the investigational drug omburtamab…
Contrary to simply stopping tumor growth, the biomedical engineers at Duke University have successfully demonstrated a new treatment for pancreatic cancer in a mouse model that may make a…
According to a story from PR Newswire, the biopharmaceutical company AMO Pharma Limited has recently announced the completion of enrollment for its REACH-CDM clinical trial. This trial will be investigating…
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