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…
In the face of COVID-19, considerations and procedures regarding the use of radiation for blood cancer patients may need to be modified. When the risk of severe infections from COVID-19…
As reported in Dravet Syndrome News, in a new study, researchers created an artificial intelligence system which could diagnose epileptic conditions more rapidly and easily than existing systems. Epilepsy is…
A 6-year-old girl presented with facial abnormalities, developmental delays, and issues with her renal system. Her family took her to their family physician. Then, when that uncovered no cause,…
As the world grows more technologically advanced, so does the medical field. Doctor-patient communication can happen over the phone or email. Telemedicine abounds. More technology is being pointed towards remote…
Kristie DeMarco was an extreme athlete- constantly training for triathlons, ironman races, ice swimming, and open water swimming. She loved to compete in various feats of strength and endurance. Overall,…
A recent study published in Blood Cancer Journal has established a new method for effectively predicting survival outcomes for patients with low-risk myelodysplastic syndromes (MDS). The method is known as the…
May 16th is celebrated annually as Hereditary Angioedema (HAE) Awareness Day. The event was established with the goal of spreading awareness about this rare condition among both the general public…
May 16th is recognized as World Congenital Disorders of Glycosylation (CDG) Day. First launched in 2016 by the Portuguese Association for CDG with support from a variety of CDG patient…
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