According to Medical XPress, Australian researchers have discovered a new and more effective way to detect remaining cancerous tissue in patients with breast cancer during surgery. This technique will assist…
In a post on the Metachromatic Leukodystrophy (MLD) Foundation's website, the organization discusses the possibilities of using gene therapy as a treatment for this rare genetic disorder. The page discusses…
According to a story from Hematology Advisor, the drug luspatercept (marketed as Reblozyl) recently demonstrated its ability to reduce transfusion burden in patients with beta thalassemia that depend on regular…
As COVID-19 continues to spread, more and more people are wondering how they might be affected. If you're a member of the rare disease community, you know it can be…
As noted recently in an article published by Cancer Network, the FDA has cleared CYNK-001 for use in COVID-19 patients. When the investigational new drug (IND) application is submitted to…
Due to the current COVID-19 pandemic, it is very difficult for people to visit their doctors. For some, this is not the biggest deal; they can stand to miss an…
According to a story from Live Science, the ongoing coronavirus/COVID-19 pandemic has upended daily life in a myriad of ways and can make leaving the house feel a little intimidating.…
Alex Biagi was planning to try out for his high schools basketball team, and could already dunk. He said his dream was to be a professional basketball player. He grew…
According to an article in the Asco Post, the FDA granted accelerated approval to Merck's KEYTRUDA®, pembrolizumab, for the treatment of metastatic small cell lung cancer (SCLC). The drug targets…
As reported in Biospace, Fulcrum Therapeutics has announced it will be submitting an application to the FDA for FTX-6058 as an Investigational New Drug (IND) by the end of 2020…
On Wednesday April 22nd, 2020, the World Orphan Drug Congress held a one hour webinar that was focused on how the rare disease community could respond to the ongoing coronavirus/COVID-19…
Según una historia de Malvern Daily Record, la compañía biofarmacéutica Millendo Therapeutics, Inc. publicó recientemente datos de su ensayo clínico de fase 2b. Este estudio estaba probando la terapia experimental…
Como se informó en Chicago Reader; a veces, la única forma de lidiar con horrores de salud insuperables es reír. Al menos así es como lo manejó Ben Byer, ya…
According to Psychology Today, French physicians may have discovered new early symptoms of COVID-19: dyshydrosis and urticaria. Though the article is published in French, you can read the dermatologists'…
According to a story from Biotech-365, the biopharmaceutical company Inventiva has been forced to change its plans to announce the latest updates for its phase IIb clinical trial. This clinical…
According to Fabry Disease News, patients with Fabry disease may have better outcomes when treated with enzyme replacement therapy PRX-102 over Replagal. In a one year comparison, PRX-102 seems…
According to MedPage Today, a recent study performed by researchers from the Vasculitis and Glomerulonephritis Center of Massachusetts General Hospital and Harvard Medical School found that patients with vasculitis had…
As reported in the CCA Blog, COVID-19 is reminding everyone what self-care means. When we can't rely on turning outwards to seek joy and comfort and good health, we must…
Ionis Pharmaceuticals, in a partnership with Roche, has announced that the enrollment has been completed for the third phase of GENERATION HD1, a trial intended to study the safety and…
COVID-19 is difficult for many of us. We are facing an unprecedented time and questions abound. How do we handle our work, our families, our health? If you are a…
The ME Association has recently published its latest weekly "research roundup" for the week of April 17th. This includes the latest research related to myalgic encephalomyelitis (ME), also known as…
According to Bronchiectasis News, patients with both HIV and bronchiectasis often have a difficult time being diagnosed with the respiratory condition. After researchers examined data from 14 patients, they discovered…
According to MedPage Today, carriers of the gene variation APOE4 are much more at risk of developing Alzheimer's disease. However, researchers found that one copy of the Klotho gene mutation,…
For Mallory Holloway, precautions to avoid lung infections have been a part of her daily life for a long time. This is because she lives with cystic fibrosis, a rare…
Happy day 134 of quarantine! Today, we're highlight four articles. First, to lift your spirits, we have a story of a young boy who made a recovery from a rare…
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