Congenital Adrenal Hyperplasia

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WHAT THE C@H?!: How This Educational Initiative Supports the Congenital Adrenal Hyperplasia (CAH) Community

Post author:Jessica Lynn
Post published:October 17, 2024
Post category:Congenital Adrenal Hyperplasia

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…

Editor’s Choice: Developments In Beta Thalassemia, Eosinophilic Esophagitis and Congenital Adrenal Hyperplasia

Post author:Colby Rogers
Post published:May 20, 2022
Post category:Congenital Adrenal Hyperplasia

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…

Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)

Post author:PW Collaborator
Post published:May 16, 2022
Post category:Congenital Adrenal Hyperplasia Rare Disease

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,…

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Mother of Twins Raises CAH Awareness

Post author:Jessica Lynn
Post published:March 4, 2022
Post category:Congenital Adrenal Hyperplasia

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…

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ICYMI: First Patient Dosed in BBP-631 Trial for CAH

Post author:Jessica Lynn
Post published:February 21, 2022
Post category:Congenital Adrenal Hyperplasia

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…

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UK MHRA Approves Efmody for CAH

Post author:Jessica Lynn
Post published:July 19, 2021
Post category:Congenital Adrenal Hyperplasia

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…

Editor’s Choice: Patient Solutions, Compassionate Use and Personal Wellbeing

Post author:Colby Rogers
Post published:July 16, 2021
Post category:Congenital Adrenal Hyperplasia

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…

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BBP-631 for CAH Granted Fast Track Designation

Post author:Jessica Lynn
Post published:May 20, 2021
Post category:Congenital Adrenal Hyperplasia

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…

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11-oxyandrogens Can Be Used as CAH Biomarkers

Post author:Jessica Lynn
Post published:February 3, 2021
Post category:Congenital Adrenal Hyperplasia

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…

https://pixabay.com/en/kid-soap-bubbles-child-fun-1241817/

Partnership Will Bring Alkindi for CAH to China

Post author:Jessica Lynn
Post published:February 1, 2021
Post category:Congenital Adrenal Hyperplasia

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…

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Phase 2b Prader-Willi Syndrome Trial Ends in Disappointment

Post author:James Moore
Post published:April 13, 2020
Post category:Congenital Adrenal Hyperplasia Prader-Willi Syndrome

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…

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Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia

Post author:James Moore
Post published:October 21, 2019
Post category:Congenital Adrenal Hyperplasia Rare Disease

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…

Potential Congenital Adrenal Hyperplasia Drug Treatment Clears Hurdle

Post author:Jean Martell
Post published:September 20, 2019
Post category:Congenital Adrenal Hyperplasia

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…

Multigenerational Mitochondrial Disease/Mitochondrial Disease Awareness

Post author:Gina Baker
Post published:September 15, 2019
Post category:Congenital Adrenal Hyperplasia Mitochondrial Disease

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…

This Young Man Accurately Diagnosed His Own Disorder But Doctors Did Not Believe His Diagnosis

Post author:Rose Duesterwald
Post published:August 6, 2019
Post category:Acute cholinergic dysautonomia Congenital Adrenal Hyperplasia Dysautonomia Hereditary sensory and autonomic neuropathy type 3 (Familial Dysautonomia)

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…

An Addison’s Disease Story: Multiple Rare Diagnoses Often Means a Longer Waiting Game

Post author:Trudy Horsting
Post published:June 27, 2019
Post category:Addison's Disease Central precocious puberty Congenital Adrenal Hyperplasia

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…

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Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial

Post author:James Moore
Post published:March 28, 2019
Post category:Congenital Adrenal Hyperplasia Rare Disease

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…

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The FDA has Awarded Over $18 Million for Rare Disease Research

Post author:Patient Worthy Contributor
Post published:September 28, 2018
Post category:Bronchopulmonary Dysplasia Congenital Adrenal Hyperplasia Cushing Disease Primary sclerosing cholangitis Stargardt Disease

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,…

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Chin Hairs in Women Could be Indicators of Cushing Syndrome or Congenital Adrenal Hyperplasia

Post author:James Moore
Post published:June 7, 2018
Post category:Congenital Adrenal Hyperplasia Cushing Syndrome Polycystic Ovary Syndrome

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…

Writer Shares Her Life Experience with Rare Congenital Adrenal Hyperplasia

Post author:Samuel Sachs
Post published:February 9, 2018
Post category:Congenital Adrenal Hyperplasia

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…

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This Experimental Drug Just Received Orphan Drug Designation For Congenital Adrenal Hyperplasia

Post author:James Moore
Post published:January 11, 2018
Post category:Congenital Adrenal Hyperplasia

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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WHAT THE C@H?!: How This Educational Initiative Supports the Congenital Adrenal Hyperplasia (CAH) Community

Editor’s Choice: Developments In Beta Thalassemia, Eosinophilic Esophagitis and Congenital Adrenal Hyperplasia

Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)

Mother of Twins Raises CAH Awareness

ICYMI: First Patient Dosed in BBP-631 Trial for CAH

UK MHRA Approves Efmody for CAH

Editor’s Choice: Patient Solutions, Compassionate Use and Personal Wellbeing

BBP-631 for CAH Granted Fast Track Designation

11-oxyandrogens Can Be Used as CAH Biomarkers

Partnership Will Bring Alkindi for CAH to China

Phase 2b Prader-Willi Syndrome Trial Ends in Disappointment

Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia

Potential Congenital Adrenal Hyperplasia Drug Treatment Clears Hurdle

Multigenerational Mitochondrial Disease/Mitochondrial Disease Awareness

This Young Man Accurately Diagnosed His Own Disorder But Doctors Did Not Believe His Diagnosis

An Addison’s Disease Story: Multiple Rare Diagnoses Often Means a Longer Waiting Game

Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial

The FDA has Awarded Over $18 Million for Rare Disease Research

Chin Hairs in Women Could be Indicators of Cushing Syndrome or Congenital Adrenal Hyperplasia

Writer Shares Her Life Experience with Rare Congenital Adrenal Hyperplasia

This Experimental Drug Just Received Orphan Drug Designation For Congenital Adrenal Hyperplasia

Metastatic Breast Cancer: Navigating Grief

Rethinking What It Means to Live With Acromegaly

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

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