According to a story from abc.net.au, Todd Murfitt from Adelaide, Australia, was only 35 when he noticed that his left hand was starting to tremor as he typed. After a…
Stargardt Disease Stargardt disease is a rare condition that causes vision loss due to the buildup of vitamin A byproducts in the retina. The byproduct causing the largest buildup is…
According to a story from globenewswire.com, the genetic medicines company Homology Medicines, Inc. has recently announced that it has opened enrollment for its phase 1/2 clinical trial. This clinical trial…
According to a story from The ASCO Post, recent studies suggest that around 70,000 adolescents and young adults are diagnosed with some type of cancer in the US each year.…
According to a story from Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This trial…
According to a story from mdmag.com, a recent study that was presented at the 2019 meeting of the American Society of Clinical Oncology has demonstrated the potential of a two…
According to a publication from The Portland Press Herald, Judge Ursula Ungaro, a senior District Judge in the Southern District of Flordia, recently ruled in favor of the United States'…
According to a story from Acrofan, the biopharmaceutical company SpringWorks Therapeutics, Inc. has recently announced the that company's experimental product candidate PD-0325901 has earned Fast Track designation from the US…
According to a story from Australasian Science Magazine, the dawn of the gene therapy "revolution" is upon us. While there are only a handful of gene therapies that have actually…
A recent study published in PLOS Biology has shown a promising new vulnerability within human viruses which could lead to the development of a new antiviral drug. This study was…
About a month ago, we reported on a story about an Irish family that is facing deportation from Australia. The family includes Anthony and Christine Hyde along with their three…
According to a story from Scleroderma News, a team of researchers associated with Michigan Medicine has been awarded a grant to the tune of $10.2 million from the National Institutes…
Transfusion-Dependent β-Thalassemia (TDT) Transfusion-dependent β-thalassemia (TDT) is a rare disease that is caused by a mutated β-globin gene. This mutation causes the amount of hemoglobin in the body to be…
According to a press release from Danish biopharmaceutical company Orphazyme, the Company intents to proceed with a marketing authorisation application (MAA) for its experimental Niemann-Pick disease type C treatment arimoclomol.…
Did you know that people who have had cancer before are more likely to get a second cancer than people who have never had cancer at all? Cancer survivors…
According to a story from Market Screener, the drug developer Reviva Pharmaceuticals, Inc. recently announced that it has completed a recent meeting with officials from the US Food and Drug…
Hereditary Hemochromatosis Hereditary hemochromatosis (HH) is a rare disease caused by hepcidin deficiency or hepcidin insensitivity. Hepcidin naturally regulates iron absorption/distribution in the body. Without hepcidin, HH patients suffer from…
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The FDA’s “untitled letter” to R3 Stem Cell (R3), based in Scottsdale Arizona, cautioned R3 that the product they are marketing is considered a drug. It must, therefore, be…
According to a story from home.cableone.net, the biopharmaceutical company Takeda Pharmaceutical Company Ltd. recently announced the latest updates to their phase 3 clinical trial which is testing the company's drug…
With the advent of a majority conservative Supreme Court with the appointment of Brett Kavanaugh last year, several US states have recently passed laws that effectively ban abortions. Many of…
According to a story from the Madison Area Lyme Support Group, one of the most difficult challenges about Lyme disease is getting a confirmed diagnosis. Testing for Lyme disease is…
According to a story from BBC, 35 year old Kimberly Chard knows that she probably doesn't have much longer to live. Kimberly was born with cystic fibrosis, a rare disorder…
According to a publication from BNN Bloomberg, regulatory authorities in the European Commission recently granted conditional marketing authorization (CMA) to Zynteglo. Zynteglo is a gene therapy designed to treat beta…
No one ever likes to think about worst possible scenarios. But sometimes by thinking about these things for a small period of time, we can make simple preparations which could…
Friedreich's Ataxia Friedreich's ataxia (FRDA) is a rare disease which is caused by a frataxin deficiency. This deficiency results in the degeneration of nerves within the spinal cord. This nerve…
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