James Dahlman, a scientist and professor at both Emory University and the Georgia Institute of Technology, has partnered with the CMT Research Foundation. The aim of this partnership is to…
A new study published in JAMA Oncology has found that of all patients who have had cancer spread to their bones and started treatment, 2,8% have developed osteonecrosis within their jaw, also…
A recent study has illuminated the need for further research into how different forms of exercise may provide certain benefits for patients living with Charcot-Marie-Tooth disease (CMT). This study was…
Sarcoma Sarcoma is a cancer that accounts for 1% of all adult cancers. It can affect individuals of any age and can impact any part of the body. It grows…
The FDA has just announced their approval of Evkeeza as a therapy for those living with homozygous familial hypercholesterolemia. The therapy is owned by Regeneron Pharmaceuticals. It was evaluated with…
There is a new collaboration in the rare cancer space which could lead to improved therapies for individuals living with these conditions. Sheperd Therapeutics and Oncoheroes Biosciences have announced that…
In a recent study, researchers have uncovered a second gene located within the mitochondria that is associated with Charcot-Marie-Tooth Disease (CMT). This study was recently published in Annals of Neurology. CMT…
A woman living with Charcot-Marie-Tooth Disease type 2 (CMT2) has experienced symptom improvement following therapy with an anti-inflammatory compound found in green tea. This study was recently published in Medicina.…
Lysogene has just announced that the FDA has approved their Investigational New Drug Application (IND) for a therapy for GM1 gangliosidosis, a rare pediatric condition. The investigational treatment is a…
A study recently published in The FASEB Journal and led by Dr. Chiharu Nishiyama from Tokyo University of Science has demonstrated that a compound found in blueberries could serve as a…
Arteriovenous malformation (AVM) is a rare disease that causes blood vessels to clump together within the skull. It can lead to aneurisms, strokes, or early death. However, surgery is possible…
Every year for the past six years the National Organization for Rare Disorders (NORD) has published a State Report Card on the progress of rare disease policies in the states.…
Microcephaly is caused by a depletion in neural progenitor cells (NPCs). This depletion is caused by a mutation in the centrosome genes, and it occurs while the brain is still…
Poly (ADP-Ribose) polymerase inhibitors (PARP inhibitors) are used to treat neoplasms. They are frequently used as a therapy for ovarian cancer. A recent study has attempted to detect the possibility…
A study has recently highlighted the fact that patients who have type 2 diabetes are more susceptible to chronic kidney disease (CKD), and as a result, cardiac issues. This study…
The very first patient has just been dosed in Pfizer's Phase 3 clinical trial for Duchenne muscular dystrophy (DMD). The trial is called CIFFREO. This patient received the therapy at a…
Mitochondrial Disease Mitochondrial disease is a rare and progressive condition that currently does not have a cure. The mitochondria is responsible for providing energy to the body. This energy is used…
A recent study has found that there is an increased risk of developing recurring thrombosis for cancer patients who have a history of venous thromboembolism, or VTE. The results of…
Parkinson's Disease Parkinson's disease is a condition which affects the nervous system. The progressive disease influences movement, and can lead to a wide array of symptoms. These may include- Tremors Stiffness…
Illumina has just formed a new partnership with Emedgene. Emedgene is an AI company which works to automatically interpret and collect data for rare genetic diseases. This data will be…
Growth differentiation factor 15 (GDF15) is expressed in normal conditions but is overly expressed when cells are under stress. It has multiple roles in different diseases and has been used…
VeDA Vestibular Disorders Association (VeDA) is a nonprofit organization that was founded in 1985. Its goal is to raise awareness of vestibular conditions, improve early diagnosis, and advocate for more efficacious…
Diagnosing Rare Diseases Diagnosing rare diseases is historically difficult. Physicians are taught to think about horses instead of zebras because horses are abundantly more common. For instance, if you're tired…
Takeda has just announced that their application to the the FDA for their eosinophilic esophagitis (EoE) investigative therapy has received Priority Review. This therapy is called budesonide oral suspension or…
Ryan Kress was crowned Ms. Wheelchair Virginia 2020 and Camille Schrier was crowned Miss America 2020. Besides their titles, what brings these two individuals together is their medical diagnosis with…
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