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…
A new treatment for Duchenne muscular dystrophy (DMD) has recently been approved by the FDA. The therapy is called viltolarsen and it was created by Nippon-Shinyaku and the National Center…
A recent study has found a 12.5% improvement for patients with severe COVID-19 when baricitinib was used in combination with remdesivir. That means a faster recovery time for patients and…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently announced that it has awarded grant funding that will go towards six clinical trials that will…
Recently, clinical-stage biopharmaceutical company Imara Inc. ("Imara") announced that the first patient was dosed in the Phase 2b Forte clinical trial. The trial is evaluating IMR-687, a PDE9-inhibitor, for…
by Lauren Taylor from In The Cloud Copy Paroxysmal nocturnal hemoglobinuria (PNH) is a very rare disease of the blood in which the affected individuals red blood cells break apart.…
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.…
by Lauren Taylor from In The Cloud Copy Latent autoimmune diabetes in adults (LADA) is a less often talked about form of diabetes that occurs as the result of the…
According to a story from BioSpace, a recent study conducted by the biotechnology company Goldfinch Bio in collaboration with academic partners is highlighting the importance of proteinuria in determining outcomes…
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…
As reported in Biospace, the National Organization for Rare Disorders (NORD) has just opened registration for patients to take part in a natural history study on metachromatic leukodystrophy (MLD), a…
by Lauren Taylor from In The Cloud Copy Polycystic ovarian syndrome or PCOS is a disorder in which women of reproductive age experience irregular menstrual cycles and/or excess androgen (a…
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…
The United States FDA has just announced that they will be providing 6 new grants, which are worth 16 million dollars for rare disease research. These grants will last a…
EB Research Partnership (EBRP) is based out of New York, United States. EB Research Foundation (EBRF) is an organization based out of Australia with the same goals. The two organizations have…
On October 14th, 2020, the International Waldenström's Macroglobulinemia Foundation (IWMF) hosted a webinar featuring Dr. Irene Ghobrial of the Dana Farber Cancer Institute. The focus of her presentation was on…
One of the biggest issues that many individuals fighting severe COVID-19 face is an overactive immune response. In effort to protect itself, the body creates dangerous levels of inflammation. Then,…
A team of scientists have created a nanoparticle tool which allows scientists across the country to study COVID-19 without any risk of infection. This particle mimics how the virus infects…
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…
Researchers have just uncovered 30 new genes that may cause Charcot-Marie-Tooth Disease (CMT). These genes were uncovered using computer analyses of biological data, a process called bioinformatics. This study was published…
A recent study published in BMC Rheumatology has found that a combination therapy could have a benefit for individuals diagnosed with diabetes and ankylosing spondylitis (AS), psoriatic arthritis (PsA), or rheumatoid arthritis…
Tarsius Pharmaceuticals has just announced positive results for their recent trial for uveitic glaucoma. This trial was called GADOT 20/20 and it was investigating a therapy called TRSo1. Ocular Diseases…
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…
According to a recent article in BioSpace, young children and infants who have been diagnosed with primary hyperoxaluria type 1 (PH1) are often faced with the use of gastrostomy…
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