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 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…
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.…
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 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…
Systemic Sclerosis Systemic Sclerosis (Systemic Scleroderma) is a rare disease which results in the hardening of the connective tissues in the body as well as the skin. It can affect…
The PoppyPocket The PoppyPocket is a wearable pocket, created to make wearing infusion pumps and well as other medical devices easier, safer, and more comfortable. It comes with two pockets,…
According to a story from Business Wire, the drug developer Cadent Therapeutics recently announced that its investigational drug CAD-1883 has been awarded Orphan Drug Designation from the US Food and…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Anavex Life Sciences Corp. has just announced that the first patient has been dosed in its phase 2 clinical trial, which…
An Idea Sometimes life leads us down unexpected paths. Mark Greget founded a medical device distribution company. That was his plan. But after interacting with patients through his work, he…
The Beginning of the Journey Claudia Martinez is a medical student at UTHealth McGovern Medical School. Her dream is, and always has been, to be a doctor. But an unexpected…
Happy Thursday! Today, we're highlighting a piece from a PW contributor sharing why it's important for people in the rare disease community to vent sometimes. If you'd like to use…
According to a story from Market Screener, the biotechnology company BioMarin Pharmaceutical Inc. recently announced that the Chinese government has approved the company's drug elosulfase alfa (marketed at Vimizim) as…
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