Evrysdi, a treatment for spinal muscular atrophy (SMA), has started to make progress around the world in terms of approval. Brazil's regulatory agency, National Health Surveillance Agency, has approved the…
A rare disease diagnosis is often shocking, and it means making life changes. An adrenoleukodystrophy (ALD) diagnosis is no exception. Because this condition typically impacts children, parents have to make…
Medical professionals have been studying inflammatory bowel disease (IBD) for many years in an effort to fully understand it. Now there has been a breakthrough, as researchers from the Children's…
Medical professionals are constantly looking for treatments that will halt or even reverse the progression of Alzheimer's disease, and a new vaccine called E22W42 may be a large step in…
Not all treatments work for every patient, with rheumatoid arthritis (RA) medications being the perfect example. Disease-modifying anti-rheumatic drugs (DMARDs) are the standard of care for RA despite the fact…
Many rare diseases are in dire need of need of viable treatments. To fix this problem, researchers have began creating and indicating existing treatments for rare diseases. An example would…
A new treatment for Krabbe disease, PBKR03, has recently been granted the Orphan Drug and Rare Pediatric Disease designations by the FDA, according to an article in GlobeNewswire. These designations…
According to CureDuchenne, two companies have began a collaboration in an effort to create new gene therapies for Duchenne muscular dystrophy (DMD). Ultragenyx and Solid Bioscience are both using their…
Novartis has recently released interim data on iptacopan, according to a press release. The data on the C3 glomerulopathy treatment was released at the American Society of Nephrology 2020 Annual…
The FDA has officially approved remdesivir for the treatment of adults and children (over the age of 12 and weighing at least 40 kg) who have been hospitalized for COVID-19.…
Monitoring tumors is an essential part of cancer treatment; it allows doctors to make informed decisions regarding which therapies to use. For many forms of cancer, medical professionals have been…
Telehealth is something that has become common during the coronavirus pandemic. It allows people to see their physicians without stepping out of the home, drastically lowering the risk of exposure.…
Ovid Therapeutics has recently announced that it will host a webinar in an effort to educate people about Angelman syndrome and their possible treatment for it, OV101. Speakers will focus…
The road to diagnosis for a rare disease patient is often a long one. Countless doctors, appointments, and tests are typically needed to finally discover the cause behind one's symptoms.…
A study recently published in Cell Stem Cell has shown that using human stem cells in combination with medical chemistry can improve existing medications to treat genetic disorders. Dr. Alfred George Jr.…
Takeda Pharmaceuticals has recently announced the results from their open-label extension study, titled VISIBLE, which evaluates Entyvio as a treatment for ulcerative colitis. According to BioSpace, these results maintain the…
According to Healio, medical professionals from Boston Children's Hospital have conducted research that provides a better look at systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease. They looked specifically…
Amolyt Pharma has recently announced that they have dosed the first patient in their trial of AZP-3601, a treatment for hypoparathyroidism. According to GlobeNewswire, this treatment will adequately address the…
Fetal alcohol spectrum disorder (FASD) is a spectrum of disorders that can impact decision making and make individuals unable to understand the consequences of their actions. Because of this, it…
Ionis Pharmaceuticals has recently released the data from their clinical trial of IONIS-ENAC-2.5Rx, a treatment for cystic fibrosis. According to Newswire, the study showed very positive results, with healthy volunteers…
Grifols, a pharmaceutical company based out of Barcelona, Spain, has announced that their potential treatment for the novel coronavirus has entered a phase 3 trial, according to BioSpace. It is…
Living with an invisible illness can be incredibly difficult. Those around you do not understand the struggle, and they can even discount your illness. Women's Health talked to three women…
Researchers have recently conducted a study using mouse models of Charcot-Marie-Tooth (CMT) disease, titled "CMTM6 Expressed on the Adaxonal Scwann Cells Surface Restricts Axonal Diameters in Peripheral Nerves." It was…
The FDA has recently granted the Fast Track designation to AGLE-102, a treatment for dystrophic epidermolysis bullosa. This designation will allow for the treatment to reach patients at a quicker…
The FDA has recently granted the Breakthrough Therapy designation to FARXIGA, a chronic kidney disease treatment created by AstraZeneca. This designation will allow for a quicker development, which will hopefully…
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