We need controls! No, not cockpit controls or videogame controls, and no, we're not out of control. We need control participants for the Dupuytren Biomarker Discovery Pilot Study. Our goal is…
The UFC has some tough fighters in it. But 35-year-old Joe Ellenberger never thought that his most difficult opponent would be within himself. That is, his paroxysmal nocturnal hemoglobinuria (PNH),…
A gene therapy for the treatment of GM1 gangliosidosis was granted Orphan Drug Designation in April. PBGM01 seeks to treat the most severe form of this condition - infantile. Read…
As reported in Medical Xpress, the U.S. Centers for Disease Control and Prevention have listed antibiotic resistance as one of the most pressing health challenges of our time. Infection treatment…
A recent BioSpace press release shares some exciting news for patients with non-small cell lung cancer, medullary thyroid cancer, and other thyroid cancers caused by RET gene mutations. Retevmo (selpercatinib) is the…
Ali Hardy struggled with her health for a long time. In an article for The Mighty, Ali, an actress in New York City, explains that she was never able to…
Immunotherapies are in the forefront for treating autoimmune diseases and cancer. Northwestern University and the University of California San Francisco conducted a study using genes that have the potential…
Immunotherapies are in the forefront for treating autoimmune diseases and cancer. Northwestern University and the University of California San Francisco conducted a study using genes that have the potential…
Zolgensma, a gene therapy for spinal muscular atrophy (SMA), has recently been granted conditional approval by the European Commission (EC). This is extremely exciting news for those living with SMA…
In 2003, Suzan Jackson was diagnosed with myalgic encephalitis, or chronic fatigue syndrome (ME/CFS). Shortly after, she was faced with the complexity of having a chronic illness. Should she tell…
The Change in Hospitals Due to COVID-19 Between 10 and 15 percent of all hospital staff is forced to isolate due to the virus at any one time. This means…
Happy Friday! If you're observing Memorial Day, enjoy the upcoming long weekend. Going to use the time to brush up on a little rare disease news? We've got three articles…
Contact Tracing Contract Tracers are an essential component to stopping the spread of COVID-19. Essentially, it entails finding those exposed to the virus and quarantining them until they are no…
According to a story from Guru Focus, the biopharmaceutical company Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. recently announced that the Ministry of Health of Spain has cleared…
Scientists say some myelin-damaging disorders have a distinctive pathology that groups them into a unique disease entity. The white knob on the left shows demyelination of a nerve fiber end.…
While the number of cases is extremely small, a University of California-San Diego professor is urging health officials to expand alerts about the mysterious illness previously seen only in children.…
One of the goals of treatment for a rare form of non-Hodgkin lymphoma is the delay of disease progression. As reported by Cancer Updates, Research and Education (CARE), a recent…
Por Jodee Redmond de In the Cloud Copy Thermo Fisher Scientific, líder mundial en la comunidad de ciencias de la vida, ha anunciado que ha desarrollado una nueva prueba para…
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…
A recent report has documented a positive case study of a potential treatment option for one of the complications Marfan syndrome can cause. This report was published in the Journal of Oral Implantology. …
¿Son los opioides un tratamiento eficaz y frecuentemente recetado para la hemofilia? En realidad, según Hemophilia News Today, los opioides se recetan a un ritmo mucho más alto de lo…
As reported in PressReleasePoint, Researchers at the University of Sheffield have found a clue to the cause of a set of rare neurological disorders known as leukodystrophies- an issue with…
Ocala Fire Rescue Department (OFD) has just launched a "community paramedicine program." The primary aim of this new program is to help keep patients who frequently call 911 out of…
Idiopathic Pulmonary Fibrosis Progression Veracyte has just signed a deal with Yale University which will help them develop the very first test which can predict disease progression for idiopathic pulmonary…
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…
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