Patient input is extremely important when evaluating treatments for rare diseases; Charcot-Marie-Tooth disease (CMT) is no exception. In order to understand the impact of different CMT therapies, researchers from the…
Translate Bio has been moving forward with the development of their new drug candidate, MRT5005. This mRNA therapy was created to address the underlying cause of cystic fibrosis (CF), and…
According to an article in BioSpace, Dyne Therapeutics has recently announced data from preclinical trials of its FORCE platform. This program is aimed at creating treatments for myotonic dystrophy type…
Turkish citizens are putting pressure on their government in regards to access to rare disease treatments, specifically spinal muscular atrophy (SMA) therapies. There are currently two drugs available to Turkish…
Landon Groves was diagnosed with Kabuki syndrome, a rare multisystem disorder, and leukemia soon after his birth. Because of these conditions, his family faces hefty medical bills. His community has…
According to BioSpace, recent research has demonstrated that CRISPR gene editing could be the answer to treating progeria, or Hutchinson-Gilford progeria syndrome. The Broad Institute and NIH have collaborated to…
Hayden Grosvenor is used to spending time in the hospital. He has Langerhans cell histiocytosis (LCH), a rare condition that is characterized by an excess of white blood cells. Because…
Grant Bonebrake was diagnosed with Alport syndrome at age 12. Since then, he has gone on to advocate for not only Alport syndrome patients, but the rare disease community as…
Monica and Josh Poynter have recently adopted a nine-year-old boy, Trey, from China. They felt a connection to the boy as he has hemophilia type A, the same rare bleeding…
The FDA has recently cleared an Investigational New Drug (IND) application for FBX-101, allowing it to move into a Phase 1/2 clinical trial. This gene therapy was developed for the…
Many people have questions about the COVID-19 vaccine, a number of them pertaining to the speed at which it was developed and approved. Rare disease patients are no exceptions. Barbra…
12-year-old Aidan Carter has Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), a rare, progressive condition that impacts mental development, appearance, physical abilities, and organ function. In order…
Cancer screenings are very important for early detection, which can then lead to treatment administered earlier in disease progression and better outcomes. Pancreatic cancer is no exception. In fact, this…
The COVID-19 pandemic has been difficult for everyone; we've all had to make changes to our lives because of it. Some of us have had to make bigger changes than…
The Neurological Alliance has released a report that details the experiences that rare, neurological disease patients in the UK share and calls for improvements for these people. Experts in the…
Landos Biopharma has recently released data from their Phase 2 trial of BT-11, a treatment for ulcerative colitis. The results were positive, making this therapy a possible viable treatment for…
A study published in BMC Pulmonary Medicine focuses on the role of endothelial progenitor cells and fibrocytes in lung sarcoidosis. They specifically investigated how these cells, along with CD34 + cells, spurred…
According to GlobeNewswire, Health Canada has approved of a biosimilar, Hyrimoz, to treat ankylosing spondylitis and eight other rare conditions. Sandoz Canada is excited about this approval, as it expands…
The FDA has recently approved of GlaxoSmithKline's (GSK) treatment for lupus nephritis (LN), BENLYSTA. This therapy is the first of its kind, meant to treat systemic lupus and adults with…
Albireo has recently initiated their Phase 3 ASSERT trial, according to GlobeNewswire. This study will evaluate odevixibat as a treatment for Alagille syndrome. This therapy fits well with Albireo's mission,…
Before 1983, there were few treatments for rare diseases. Various obstacles stood in the way, including little representation for patients, few incentives, and a lack of resources. Fortunately, the Orphan…
Yash Cary knows what it's like to spend the holidays in the hospital. In fact, three of his last six Christmases have been at UNC Hospital. This is because Yash…
"Once Upon a Christmas Miracle" is a Hallmark movie, falling into their "Movies and Mysteries" holiday series. It tells the true story of Heather Krueger and Chris Dempsey, a couple…
According to an article from Cancer Therapy Advisor, medical professionals have created a new, simplified prognostic score for patients affected by myelodysplastic syndromes (MDS). This score utilizes multiparametric flow cytometry…
Selma Santos has met many nurses over the course of her life. Having a daughter with cystic fibrosis means spending a lot of time in the hospital or at doctor's…
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