A gene therapy for the treatment of GM1 gangliosidosis was granted Orphan Drug Designation in April. PBGM01 seeks to treat the most severe form of this condition - infantile. Read…
As reported in Medical Xpress, the U.S. Centers for Disease Control and Prevention have listed antibiotic resistance as one of the most pressing health challenges of our time. Infection treatment…
A recent BioSpace press release shares some exciting news for patients with non-small cell lung cancer, medullary thyroid cancer, and other thyroid cancers caused by RET gene mutations. Retevmo (selpercatinib) is the…
Ali Hardy struggled with her health for a long time. In an article for The Mighty, Ali, an actress in New York City, explains that she was never able to…
Immunotherapies are in the forefront for treating autoimmune diseases and cancer. Northwestern University and the University of California San Francisco conducted a study using genes that have the potential…
Immunotherapies are in the forefront for treating autoimmune diseases and cancer. Northwestern University and the University of California San Francisco conducted a study using genes that have the potential…
Happy Friday! If you're observing Memorial Day, enjoy the upcoming long weekend. Going to use the time to brush up on a little rare disease news? We've got three articles…
While the number of cases is extremely small, a University of California-San Diego professor is urging health officials to expand alerts about the mysterious illness previously seen only in children.…
As reported by producer Minoryx; the pharmaceutical leriglitazone, used to treat early stage cerebral ALD (cALD) has just announced its plans to continue onto its Phase 2 Nexus trial without…
By Danielle Bradshaw from In The Cloud Copy 19-year-old Marky Jaquez’s feet have never touched the ground because the sensitive skin of his soles would tear away. As such, he…
For those in the rare disease community, you know that it can take a long time to reach a diagnosis for your condition: an average of 4.8 years! But upon diagnosis, many…
Disposable Scopes Disposable scopes are exactly as they sound. Medical scopes utilized for the bladder, lungs, colon, etc. can be made reusable or disposable. Disposable scopes allow faster and more…
The last decades have seen significant progress when it comes to medical care for rare diseases. Tasks doctors once slaved over were transformed with better speed and efficiency by modern…
Bridging the Voice of ALS Patients | ATSDR Bridging the Voice of ALS Patients Because learning more about ALS is an important step in the battle to defeat it, the…
Promising new drug target for autoimmune and allergic diseases revealed Credit: The Babraham Institute A study led by researchers at the Babraham Institute in collaboration with the Wellcome Sanger Institute…
Many patients with rare diseases require some sort of treatment or therapy to manage their conditions. In the case of atypical hemolytic-uremic syndrome, or aHUS, this therapy is dialysis. Learn…
By Natalie Homan from In The Cloud Copy The COVID-19 pandemic has changed the lives of millions around the world in an incredibly short span of time. It’s hard to…
By Natalie Homan from In The Cloud Copy Two businesses that advocate for patients with rare and orphan diseases announced recently that they are teaming up to help patients get…
This was originally published as the Seventh Newsletter for the Leukodystrophy Resource Research Organisation (LRRO) on April 19th 2020. You can read more from their newsletter here. To state the blatantly…
Click here to read part 1 and here to read part 2 After coming back from the Global Genes conference, Kristie was convinced that establishing a foundation was the right…
May is recognized as Behçet's Disease Awareness Month and May 20th is recognized as Behçet's Disease Awareness Day. The focus of these events is to help spread awareness about this…
According to CNN, the CDC recently sent out a health advisory to doctors across the United States alerting them of a new syndrome affecting children in eighteen states, Washington, D.C.,…
According to a story from Sanfilippo News, the healthcare tech company RDMD has recently announced that it has raised $14 million in Series A financing that will be dedicated towards…
According to a recent press release published by the National Institutes of Health (NIH), a new survey spearheaded by the Rare Diseases Clinical Research Network (RDCRN) is seeking to understand…
To read part 1 click here Kristie’s medical mystery was now solved. What was the next step? After speaking with her genetic counselor about her results, Kristie was referred to…
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