Trisha Thaens compiled this very moving video of her Cushing's journey. She says "OK, here is the link. I think it is best watched in full screen (the squarebutton in the bottom right hand corner). I wasn't going to share quite yet, but was asked to because its Rare Disease Day."
Have questions about the new Korlym? How about Korlym vs ketoconazole? About medical vs surgical treatment for Cushing's.
Ask Dr. Theodore Friedman.
Theodore C. Friedman, M.D., Ph.D. has opened a private practice, specializing in treating patients with adrenal, pituitary, thyroid and fatigue disorders. Dr. Friedman has privileges at Cedars-Sinai Medical Center and Martin Luther King Medical Center. His practice includes detecting and treating hormone imbalances, including hormone replacement therapy. Dr. Friedman is also an expert in diagnosing and treating pituitary disorders, including Cushings disease and syndrome.
Dr. Friedman's career reflects his ongoing quest to better understand and treat endocrine problems. With both medical and research doctoral degrees, he has conducted studies and cared for patients at some of the country's most prestigious institutions, including the University of Michigan, the National Institutes of Health, Cedars-Sinai Medical Center, and UCLA's Charles Drew University of Medicine and Science.
Read Dr. Friedman's First Guest Chat, November 11, 2003.
Read Dr. Friedman's Second Guest Chat, March 2, 2004.
Listen to Dr. Friedman First Live Voice Interview, January 29, 2009.
Listen to Dr. Friedman Second Live Voice Interview, March 12, 2009.
Listen to Dr. Friedman Third Live Voice Interview, February 13, 2011.
Listen live at http://www.blogtalkradio.com/cushingshelp/2012/03/13/questions-about-medical-therapies-for-cushings
Call in to ask your question at
(646) 200-0162
This interview will be archived afterwards at the same link and on iTunes Cushie Podcasts
Have questions about the new Korlym? How about Korlym vs ketoconazole? About medical vs surgical treatment for Cushing's.
Ask Dr. Theodore Friedman.
Theodore C. Friedman, M.D., Ph.D. has opened a private practice, specializing in treating patients with adrenal, pituitary, thyroid and fatigue disorders. Dr. Friedman has privileges at Cedars-Sinai Medical Center and Martin Luther King Medical Center. His practice includes detecting and treating hormone imbalances, including hormone replacement therapy. Dr. Friedman is also an expert in diagnosing and treating pituitary disorders, including Cushings disease and syndrome.
Dr. Friedman's career reflects his ongoing quest to better understand and treat endocrine problems. With both medical and research doctoral degrees, he has conducted studies and cared for patients at some of the country's most prestigious institutions, including the University of Michigan, the National Institutes of Health, Cedars-Sinai Medical Center, and UCLA's Charles Drew University of Medicine and Science.
Read Dr. Friedman's First Guest Chat, November 11, 2003.
Read Dr. Friedman's Second Guest Chat, March 2, 2004.
Listen live at http://www.blogtalkradio.com/cushingshelp/2012/03/13/questions-about-medical-therapies-for-cushings
Call in to ask your question at
(646) 200-0162
This interview will be archived afterwards at the same link and on iTunes Cushie Podcasts
This is a "knol" that I wrote in 2008. It was accepted by the Open Journal of Medicine.
Now that "knols" are being discontinued, the Open Journal moved this to their site but all of the images were lost in the move. Also, the name of one of their authors was added to my knol.
I'm posting this here to keep the information as it was, not as it has morphed into.
MENLO PARK, Calif. - February 17, 2012
Corcept Therapeutics (NASDAQ:CORT) announced today that the U.S. Food and Drug Administration (FDA) has approved Korlym™ (mifepristone) 300 mg Tablets as a once-daily oral medicine to control hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing's syndrome who have diabetes mellitus type 2 or glucose intolerance and have failed surgery or are not candidates for surgery.
"We appreciate the FDA's diligent attention to our NDA and its grant of approval on the PDUFA date," said Joseph K. Belanoff, M.D., the company's Chief Executive Officer. "We plan to make Korlym available to patients by May 1 through a distribution system designed to support both patients and prescribers."
Corcept will be the sole marketer of Korlym. "A relatively small number of endocrinologists regularly treat patients with Cushing's syndrome," added Dr. Belanoff. "These doctors can be reached without a large sales and marketing infrastructure." The company has begun hiring Medical Science Liaisons to inform practitioners about the drug, which will be dispensed by the leading specialty pharmacy company CuraScript SP, a subsidiary of Express Scripts.
"Korlym is a significant advance in the treatment of patients suffering from the debilitating symptoms of Cushing's syndrome," said Robert L. Roe, M.D., Corcept's President. "For the first time, these patients have access to an approved therapy when surgery has failed or is not an option."
Korlym clinical trial investigator Amir Hamrahian, M.D., Department of Endocrinology, Diabetes and Metabolism at the Cleveland Clinic said, "There are not many effective treatment options for patients with Cushing's syndrome. Although surgery is standard first line treatment for the disease, it is not always successful and not all patients are candidates. As part of the clinical trial, I have used Korlym successfully and my patients continue to do well on the medicine. I'm excited to be able to continue using Korlym in these patients and others who need it. This medicine's approval gives me a much needed tool to better treat patients."
Dr. Hamrahian's comments were seconded by Maureen V., a patient in Corcept's Phase 3 clinical trial: "I had pituitary surgery to treat my Cushing's syndrome. Unfortunately, my surgery wasn't successful. I was lucky to get into the study and get Korlym treatment. I have been taking the medicine successfully for over a year, and I am extremely happy that it was approved by the FDA. Now I know I'll be able to keep taking it. It has made a big difference in my life."
Clinical Trial Results Supporting FDA Approval
The clinical data supporting the FDA approval of Korlym resulted from an uncontrolled, open-label, multi-center, 24-week phase III study of 50 patients who had endogenous Cushing's syndrome and were either not eligible for or had relapsed from surgery and were either glucose intolerant (29 patients) or had hypertension (21 patients). Within the glucose intolerant group, 60 percent of patients had a greater than 25 percent reduction from baseline in the area under the curve in the oral glucose tolerance test. In this group, mean hemoglobin A1C (HbA1C) was reduced from 7.4 percent to 6.3 percent. All 14 patients with above-normal HbA1C levels at baseline experienced reductions. Eight of these 14 normalized their HbA1C. Antidiabetic medications were reduced in seven of the 15 patients with diabetes mellitus type 2 and remained constant in the others.
Patients who responded to therapy were allowed enrollment in an extension trial. Eighty-eight percent of the patients who completed the trial chose to do so.
A peer-reviewed analysis of the study results will soon be published in a leading journal.
Patients in the study started Korlym treatment on a dose of 300 mg administered once daily. Their dose was then titrated to maximum clinical effect. As indicated in the medicine's label, physicians prescribing Korlym may determine the appropriate dose for each patient by assessing tolerability and degree of improvement in Cushing's syndrome manifestations. In the first six weeks, these manifestations may include changes in glucose control, anti-diabetic medication requirements, insulin levels and psychiatric symptoms. After two months, assessment may also be based on improvements in cushingoid appearance, acne, hirsutism, striae, decreased body weight, along with further changes in glucose control.
About Korlym™ (mifepristone) 300 mg Tablets
Korlym is a once-daily oral medication that blocks the glucocorticoid receptor type II (GR-II) to which cortisol normally binds. By blocking this receptor, Korlym inhibits the effects of excess cortisol in Cushing's syndrome patients.
The FDA has designated Korlym as an Orphan Drug for treatment of the clinical manifestations of endogenous Cushing's syndrome. Orphan Drug designation is a special status designed to encourage the development of medicines for rare diseases and conditions. Because Korlym is an Orphan Drug, Corcept will have marketing exclusivity consistent with the FDA's designation until February 2019.
About Cushing's Syndrome
Endogenous Cushing's syndrome is a rare and life-threatening endocrine disorder that results from long-term exposure to excess levels of the hormone cortisol. This excess is caused by tumors that usually occur in the pituitary or adrenal glands that over-produce, or prompt the over-production of, cortisol.
Although cortisol at normal levels is essential to health, in excess it causes a variety of problems, including hyperglycemia, upper body obesity, a rounded face, stretch marks on the skin, an accumulation of fat on the back, thin and easily bruised skin, muscle weakness, bone weakness, persistent infections, high blood pressure, fatigue, irritability, anxiety, psychosis and depression. Women may have menstrual irregularities and facial hair growth, while men may have decreased fertility or erectile dysfunction. More than 70 percent of Cushing's syndrome patients suffer from glucose intolerance or diabetes.
The treatment of an endogenous Cushing's syndrome patient depends on the cause. The first-line approach is surgery to remove the tumor. If surgery is not successful or is not an option, radiation may be used, but that therapy can take up to ten years to achieve full effect. Surgery and radiation are successful in only approximately one-half of all cases.
If left untreated, Cushing's syndrome has a five-year mortality rate of 50 percent.
An orphan disease, Cushing's syndrome occurs in about 20,000 people in the United States, mostly women between the ages of 20 and 50.
Conference Call Information
Corcept will hold a conference call on Tuesday, February 21, 2012 at 9:00 a.m. Eastern Time (6:00 a.m. Pacific Time) to discuss this announcement. To participate in the live call please dial 1-800-264-7882 from the United States or +1-847-413-3708 internationally. The pass code is 31838602. Please dial in approximately 10 minutes before the start of the call.
A replay of the conference call will be available through March 6, 2012 at 1-888-843-7419 from the United States and +1-630-652-3042 internationally. The pass code is 31838602.
IMPORTANT SAFETY INFORMATION
WARNING: TERMINATION OF PREGNANCYSee full prescribing information for complete boxed warning.has potent antiprogestational effects and will result in the termination of pregnancy. Pregnancy must therefore be excluded before the initiation of treatment with Korlym, or if treatment is interrupted for more than 14 days in females of reproductive potential.Contraindications
Warnings and Precautions
Adverse Reactions
Most common adverse reactions in Cushing's syndrome (≥ 20%): nausea, fatigue, headache, decreased blood potassium, arthralgia, vomiting, peripheral edema, hypertension, dizziness, decreased appetite, endometrial hypertrophy.
To report suspected adverse reactions, contact Corcept Therapeutics at 1-855-844-3270 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Drug Interactions
Use in Specific Populations
Please see the accompanying full Prescribing Information including boxed warning atwww.corcept.com/prescribinginfo.pdf
Please see the accompanying Medication Guide at www.corcept.com/medicationguide.pdf
About Corcept Therapeutics Incorporated
Corcept is a pharmaceutical company engaged in the discovery, development and commercialization of drugs for the treatment of severe metabolic and psychiatric disorders. Korlym, a first generation GR-II antagonist, is the company's first FDA-approved medication. The company has a portfolio of new selective GR-II antagonists that block the effects of cortisol but not progesterone. Corcept also owns an extensive intellectual property portfolio covering the use of GR-II antagonists, including mifepristone, in the treatment of a wide variety of psychiatric and metabolic disorders. The company also holds composition of matter patents for its selective GR-II antagonists.
Statements made in this news release, other than statements of historical fact, are forward-looking statements. Forward-looking statements are subject to a number of known and unknown risks and uncertainties that might cause actual results to differ materially from those expressed or implied by such statements. For example, there can be no assurances that clinical results will be predictive of real-world use, or regarding the pace of Korlym's acceptance by physicians and patients, the reimbursement decisions of government or private insurance payers, the effects of rapid technological change and competition, the protections afforded by Korlym's Orphan Drug Designation or by Corcept's other intellectual property rights, and the cost, pace and success of Corcept's other product development efforts. These and other risks are set forth in the Company's SEC filings, all of which are available from our website (www.corcept.com) or from the SEC's website (www.sec.gov). We disclaim any intention or duty to update any forward-looking statement made in this news release.
CONTACTS:
Invvestor ContactKorlym was approved February 17th, 2012 for persistant Cushing's. More information here.
Corcept will hold a conference call on Tuesday, February 21, 2012 at 9:00 a.m. Eastern Time (6:00 a.m. Pacific Time) to discuss this announcement. To participate in the live call please dial 1-800-264-7882 from the United States or +1-847-413-3708 internationally. The pass code is 31838602. Please dial in approximately 10 minutes before the start of the call.
A replay of the conference call will be available through March 6, 2012 at 1-888-843-7419 from the United States and +1-630-652-3042 internationally. The pass code is 31838602.
Brochures for Primary Care Physicians, to increase awareness of Cushing's syndrome. In English and Spanish
These were sent to me by a Facebook friend who wrote:
it's Vida A, I have writen in the facebook group that I would like to upload some information that I think it's very useful. Most of it is in Spanish but maybe someone is able to understand it.
I've found it in: www.ciberer.es
then you go to enfermedades raras > divulgación social > boletin social and then it's in Boletin Social number 3 (dedicated to Cushing)
I think it may be very useful
Thanks very much
Núria (Vida A)
Authors: Toja, Paola M.1; Branzi, Giovanna2; Ciambellotti, Francesca2; Radaelli, Piero3; De Martin, Martina1; Lonati, Laura Maria2; Scacchi, Massimo; Parati, Gianfranco; Cavagnini, Francesco1; Giraldi, Francesca Pecori
Source: Clinical Endocrinology, Volume 76, Number 3, 1 March 2012 , pp. 332-338(7)
Publisher: Wiley-Blackwell
Abstract:
Objectives Sustained hypercortisolism impacts cardiac function, and, indeed, cardiac disease is one of the major determinants of mortality in patients with Cushing's syndrome. The aim of this study was to assess the clinical relevance of cardiac structure and function alterations by echocardiography in patients with active Cushing's syndrome and after disease remission.
Study design Seventy-one patients (61 women, 10 men) with Cushing's syndrome and 70 age-, sex- and blood pressure-matched controls were enrolled. Echocardiography was performed in 49 patients with active disease and at several time points after remission in 44 patients (median follow-up 46·4 months), and prevalence of abnormal left ventricular mass measurements and systolic and diastolic functions indices was compared between patients with active disease, after remission and controls. Twenty-two patients were evaluated both before and after remission.
Results Up to 70% of patients with active Cushing's syndrome presented abnormal left ventricular mass parameters; 42% presented concentric hypertrophy and 23% concentric remodelling. Major indices of systolic and diastolic functions, i.e. ejection fraction and E/A ratio, respectively, were normal. Upon remission of hypercortisolism, left ventricular mass parameters ameliorated considerably, although abnormal values were still more frequent than in controls. Both cortisol excess and hypertension contribute to cardiac mass alterations and increase the prevalence of target organ damage.
Conclusions Cushing's syndrome is associated with an increased risk for abnormalities of cardiac mass, which ameliorates, but does not fully disappear after remission. Systolic and diastolic functions are largely within the normal range in these patients.
Document Type: Research article
DOI: http://dx.doi.org/10.1111/j.1365-2265.2011.04206.x
Affiliations: 1: Ospedale San Luca, Neuroendocrinology Research Lab, Istituto Auxologico Italiano IRCCS 2: Department of Cardiology, Ospedale San Luca, Istituto Auxologico Italiano IRCCS 3: Department of Medical Sciences, University of Milan
Buy this article here: http://www.ingentaconnect.com/content/bsc/cend/2012/00000076/00000003/art00004
[prMac.com] South Dartmouth, Massachusetts - The Editors of Endotext and Thyroidmanager announce the release of their new medical app, "Endocrinology and Endo Emergency".
The app provides concise, practical information on endocrine emergencies, and provides the answers needed RIGHT NOW for their care. Each of the 36 topics covered includes the clinical recognition, pathophysiology, diagnosis, differential, treatment, and follow up. In addition, references to available Clinical Guidelines, and important literature citations are provided.
Focusing on the critical information needed at the point of care, the app includes topics such as Cushing's disease, diabetic ketoacidosis, managing acute type I and type II diabetes, hypercholesterolemia, adrenal insufficiency, hyperthyroidism, thyroid storm, and much more. Key facts are presented in tables and flow-charts to allow quick reference to crucial information.
The app is designed for physicians, nurses, emergency room caregivers, hospitalists, trainees, students, and any caregiver who needs to treat seriously ill patients.
The "Endocrinology and Endo Emergency" mobile application was created by the world-renowned endocrinologists who also brought you ENDOTEXT.ORG and THYROIDMANAGER.ORG, the most comprehensive and up-to-date web-based endocrine textbooks. Endocrinology and Endo Emergency is always updated with the latest information, and updates can be automatically downloaded by users.
To view or purchase the app in the Apple App store (also available for android) or go to endoemergencies website for information and to view a sample section. Qualified reviewers may contact us for promo codes.
* Covers endocrinology and endocrine emergencies
* Provides critical information needed at the point of care
* 36 topics covered
* written by expert endocrinologists from around the world
Device Requirements:
* iPhone 3GS, iPhone 4, iPhone 4S, iPod touch (3rd generation), iPod touch (4th generation) and iPad
* Requires iOS 5.0 or later
* 1.7 MB
Endocrine Emergencies
Thyroid Disease Manager
Endotext Web Book
Purchase and Download
Screenshot
App Icon
Produced and owned by Endocrine Education, Inc. and Mdtext.Com, Inc. Copyright (C) 2012 Endocrine Education, Inc. and Mdtext.Com, Inc. All Rights Reserved. Apple, the Apple logo, iPhone, iPod and iPad are registered trademarks of Apple Inc. in the U.S. and/or other countries.
Update: Wednesday, February 8, 2012 is the last day to call.
We invite you to help us create better information, tools, and resources
for people with Cushing’s Syndrome
How: Share your opinions and give input in a one-on-one confidential interview. Your opinions will remain confidential. The overall results will help others with Cushing’s Syndrome.
When: Market research interviews can be conducted by phone or in-person, in a location near you. The interview will last approximately 45 minutes.
If you are interested in a phone interview or in-person interview, please contact Clair Carmichael Johnstone (see details below). More information (including cities and locations for in-person interviews) will be provided on the phone.
Am I Eligible? If you’ve been diagnosed with Cushing’s Syndrome and still experience symptoms of Cushing’s you are eligible to participate. Cushing’s Syndrome includes: Cushing’s Disease, ectopic Cushing’s and adrenal Cushing’s. Patients should have been diagnosed within the last 10 years.
At this time, patients who are in remission, were diagnosed more than 10 years ago, or have had a bilateral adrenalectomy are not eligible for research.
Details: Participants eligible for market research will be asked to participate in the 45-minute interview. Patients participate also receive an honorarium (payment) for your time. Those who participate in an in-person interview will be compensated $250 and those who choose a phone interview will receive $125 for their time.
Why? Corcept Therapeutics is interested in hearing from people who suffer from Cushing’s Syndrome in order to improve treatment and information available. This can lead to improved education and resources for those with Cushing’s and physicians treating Cushing’s.
How do I find out more?
If you have questions or would like to participate, please contact Clair Carmichael Johnstone at:
Toll-free number: (800) 856-6706, or
E-mail: cushing@compasshc.com
Please provide your name, phone number, and the best times to reach you so we can follow up promptly.
