A recent preprint reported by medRxiv explores the complex relationship between genetic mutations and clinical presentation in Colombian patients with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD). While…
For over three decades, Neurocrine Biosciences, Inc. has been working to support individuals with neurological, neuroendocrine and neuropsychiatric disorders through the development of breakthrough treatment options. One of the company’s…
Happy Friday! This week, we have details on an investigational compound for beta thalassemia. Also, we break down the details of a new study that examines psychiatric illness rates among…
Acknowledgment: This story is sponsored by Neurocrine Biosciences and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…
When Rachel Matthews birthed her twin boys, Aiden and Gavin, she was ecstatic. But just a day or two after Rachel had returned home with her boys, she received a…
In late January 2022, biopharmaceutical company BridgeBio Pharma, Inc. ("BridgeBio") shared via news release that the first patient had been dosed in the Phase 1/2 ADventure clinical trial. Within this…
In the United Kingdom, the Medicines and Healthcare products Regulatory Agency (MHRA) regulates medication, transfusion components, and medical devices, making sure they meet safety and efficacy standards. According to Pharma…
Happy Friday! This week, we examine the struggle to access experimental rare therapies through compassionate use. Also, in our new Compassion Corner series, we examine why addressing patients' personal wellbeing can…
According to corporate news shared on Street Insider, biopharmaceutical company BridgeBio Pharma, Inc. ("BridgeBio") is making ground with its AAV5 gene therapy, BBP-631. Recently, the FDA granted Fast Track designation…
How can researchers diagnose and study disease progression if typical biomarkers are unavailable or inconclusive? This very issue arose during a study on congenital adrenal hyperplasia (CAH). As shared in…
In 2018, China declared congenital adrenal hyperplasia (CAH) to be a rare disease, and it is estimated to affect 1 in every 6084 Chinese births. However, there are currently…
According to a story from Malvern Daily Record, the biopharmaceutical company Millendo Therapeutics, Inc. recently released data from its phase 2b clinical trial. This study was testing the company's experimental…
According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The…
Some promising news for the congenital adrenal hyperplasia (CAH) community! Spruce Biosciences is one step closer to a treatment for CAH after new phase 2a data showed the drug reduced…
This year Mitochondrial Disease Awareness week is September 15-21 but I and other members of my family will continue to fight this battle for the rest of our lives. For…
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Doug Lindsay ran track in high school. According to an article published by CNN Health, Doug had big plans as a senior at Rockhurst University in Kansas City where he…
Emily Dover This is Emily Dover's story. At birth- perfectly healthy newborn, smaller than her siblings (8 pounds) 1 week- was having trouble sleeping and began crying in pain 4…
According to a story from BioSpace, the biotechnology company Spruce Biosciences recently announced that they have achieved positive proof of concept data in the company's clinical trial. This is a…
The United States Food and Drug Administration has awarded twelve research grants worth a total of over $18 million for research into rare diseases. For more information about this news,…
According to a story from msn.com, a significant portion of woman may notice a small number of hairs growing on their chin at some point in their lives. In most…
In her recent story with Huffington Post, Allison Landa describes her experiences with congenital adrenal hyperplasia. Her article, self-titled as “My Life as a Former Bearded Lady,” details a lifetime…
According to a story from prnewswire.com, the experimental treatment SPR001 was just granted orphan drug status by the Food and Drug Administration (FDA). The new treatment is currently being developed…
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