Cystic Fibrosis Cystic fibrosis (CF) is a rare disease caused by the lack (or malfunctioning) of the cystic fibrosis transmembrane conductance regulator (CTFR) protein. This occurs when children inherit a mutated…
Neutropenia Neutropenia is a rare condition that affects approximately 1 in every 200,000 newborns. It results in a reduced number of neutrophils in the body. Neutrophils are white blood cells,…
A report published in 2018 in The Hemotologist mentions the fact that most of the therapies introduced over the years for acute myeloid leukemia (AML) simply meant optimizing the use of…
According to a story from globenewswire.com, the biopharmaceutical company Lysogene and the drug developer Sarepta Therapeutics, Inc., have recently announced that the first patient has been dosed in a Phase…
The University of Louisville’s publication UL News recently reported a finding by its research team regarding an immune checkpoint molecule. The molecule had been developed for cancer immunotherapy but when…
According to a story from BBC, Mats Steen passed away at 25 years old due to complications from his Duchenne muscular dystrophy. Upon his death, parents Robert and Trude grieved…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. This forum will take place in Jacksonville, Florida When: Saturday, March 30th, 2019 Where: Mayo Clinic Cancer…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. This forum will take place in Phoenix, Arizona. When: Saturday, March 9th, 2019 Where: Sheraton Crescent…
The Washington Post reported in December of last year that the 8-year-old Matadi Sela Petit had died during surgery in a Los Angeles hospital. The accident was caused by a…
According to a story from EurekAlert!, a new type of cancer drug that is being described as a "Trojan horse" that infiltrates cancer tumors is showing potential in several types…
Interstitial Lung Disease in Systemic Sclerosis Systemic sclerosis (SSc), sometimes referred to as systemic scleroderma, is a rare autoimmune condition. It results in the buildup of fibrosis in the skin…
Cryptococcosis Cryptococcosis is a disease caused by the Cryptococcus fungus. It can affect the lungs, the spinal cord, or the brain. When the brain is infected, the condition is called…
According to a story from metro.co.uk, celebrity Katie Price, an English television personality, author, singer, and designer is reportedly considering part time residential care for her son Harvey. Harvey has…
Hypoparathyroidism Hypoparathyroidism (HP) is caused by low levels of the parathyroid hormone (PTH). Most people develop this disease after thyroid surgery as a result of accidental damage/removal of the parathyroid…
A recent interview with David Alvarez and his mother in the CTPost explains how he manages to remain positive after being diagnosed with spinal muscular atrophy (SMA). David is a freshman…
According to a story from BioPortfolio, Takeda Pharmaceutical Company Limited recently announced that its drug ADCETRIS (brentuximab vedotin) has seen its marketing authorization extended in the European Union. The European…
In a recent article appearing in BioPortfolio, Orphan Technologies announced a Phase 1/2 clinical trial involving the first Classical Homocystinuria (HCU) patients to be treated with OT-58. Twelve patients were enrolled as…
Loukisha shares her journey with hereditary angioedma (HAE). Living with a rare disease you'll experience highs and lows. Unfortunately the moments of struggle are unavoidable; all you can do is…
According to a story from Andre Eger, the drug developer Gilead recently had the misfortune to announce that its clinical trial testing an experimental drug for late stage nonalcoholic steatohepatitis…
According to a study published in the Orphanet Journal of Rare Diseases, a study that was intended to identify a new approach to monitoring the progression of x-linked adrenoleukodystrophy in women…
Content Warning: The following article discusses sexual abuse. According to a story from Buzzfeed News, many former patients of an esteemed medical researcher named Reginald Archibald, who was affiliated with…
In order to begin clinical trials for an experimental new drug, pharmaceutical companies in the U.S. are required to submit an Investigational New Drug (IND) application to the FDA. They…
According to a story from IT Business Net, the medical company KIYATEC, Inc., has recently announced that that the Knight Cancer Institute at Oregon Health & Science University has joined…
Tuberous Sclerosis Tuberous sclerosis complex (TSC) is a rare condition which results in the buildup of tumors in the skin, kidneys, brain, as well as other organs. Although the tumors are…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is an autoimmune condition which causes inflammation throughout the digestive tract. There are two main forms of the condition- Crohn's disease and the…
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