Claire Richmond lives with acute hepatic porphyria (AHP). She searched for 19 years to find answers to her unexplained pain. She wasn't diagnosed until she was 32. She recently had…
Results from a study on systemic mastocytosis (SM) have recently been published. This investigation aimed to understand the prevalence of contrast media induced hypersensitivity reactions (CMIHR) in different subtypes of…
The FDA, the NIH, 5 nonprofits, and 10 pharmaceutical companies, have formed an official partnership to accelerate gene therapy development for rare diseases. Out of 7,000 known rare diseases, just…
The Vestibular Disorder Association (VeDA) has just published a guide to handling the holidays when you are a vestibular patient. Holidays can be a challenge because of overstimulation, fatigue, high…
Many doctors have thought in the past that age should be an automatic ineligibility factor for multiple myeloma patient transplants. However, Dr. Langren, Dr. Gasparetto, Dr. Landgren, Dr. Hofmeister, and…
IMV Inc. recently presented two posters at the AACR-NCI-EORTC Virtual International Conference on Molecular Targets and Cancer Therapeutics showcasing their DPX delivery platform's immunotherapeutic capabilities. IMV Inc. IMV Inc. is…
LogicBio Therapeutics has just presented preclinical data of their GeneRide platform. This data demonstrates that the GeneRide technology is valuable in methylmalonic acidemia (MMA), hereditary tyrosinemia type 1 (HT1), and…
Albert Edge and Yushi Hayashi recently led a research team which uncovered the specific mechanism which can cause deafness in Norrie disease, a rare condition. These findings were recently published…
The University of Pittsburgh School of Medicine along with Prairie View A&M University, have recently uncovered that there are some cancer drugs which could be used as an effective treatment…
Aurion Biotech has just released the results from their clinical trial called IOTA. This trial was conducted by researchers in Japan and demonstrated that corneal endothelial cell therapy was able…
Healx has just announced that they've been given Investigational New Drug FDA approval for HLX-0201 for Fragile X syndrome. This means they are able to move forward with their Phase 2a…
LEXEO Therapeutics and FUJIFILM Diosynth Biotechnologies have just announced their collaborative partnership. The aim is to develop and ultimately manufacture the AAV-mediated gene therapies developed by LEXEO. LEXEO LEXEO is…
The COVID-19 pandemic has led to many changes, disruptions, and setbacks. For rare diseases, it did delay research for new treatments. However, there were a few positive things that came…
A new article has been published in Neurology and Therapy which specifically discusses the lived experiences of patients with myasthenia gravis (MG). The most notable thing about this article is that…
Lyme disease is typically treated with a broad-spectrum antibiotic. Unfortunately, these can have just as many negative effects as they can positive. For instance, they can increase the chance that…
The 2nd Virtual Scientific Conference for the International Association for Chronic Fatigue Syndrome/ Myalgia Encephalomyelitis was recently held in August 2021. This conference was a place for researchers to present work which…
Dr. Bakris, Dr. Agarwal, Dr. Wright, and Dr. Minkoff have recently come together to discuss how they assess kidney function and ultimately diagnose chronic kidney disease (CKD). Assessing Disease Severity…
Three doctors recently got together to discuss how they manage care for their sickle cell disease (SCD) patients. As a part of this conversation, they discussed how they combat systemic…
Rare Diseases South Africa (RDSA) and The Rare Diseases Access Initiative (RDAI) have recently come together to talk about the issues that those living with rare diseases in South Africa…
Maya Doyle is a health social worker specifically focused in pediatrics and the transition from pediatric care to adult care. She has been practicing for twenty years. Now, she works…
The very first National Census of Patients with Rare Diseases in Mexico began October first of 2021 by the General Health Council (CSG). This census will last until January 15th…
Data has recently been announced from a study investigating the efficacy of a new therapy called Alpelisib, for the treatment of PIK3CA-related overgrowth spectrum (PROS). The study was called EPIK-P1.…
Japan's Ministry of Health, Labour, and Welfare has just announced its approval of Alofisel, a stem cell therapy created by Takeda. This approval is specifically for patients who are diagnosed…
CurePSP has just announced their latest Venture Grant recipients. These grants total $320,000 and will all support the research of neurodegenerative disease treatments. CurePSP gives these grants twice every year.…
Emmalyn Reese Hudson was born 11 years ago at 35 weeks gestation weighing in at 5 lbs, 7 ozs. and 18 inches long. Our beautiful baby girl was so tiny…
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