RARE Webinar! Learning More on Informed Consent

Posted on November 5, 2015 by MaryO
a doctor in his office showing an informed consent document and pointing with a pen where the patient must to sign

a doctor in his office showing an informed consent document and pointing with a pen where the patient must to sign

 

Wednesday, November 18, 2015 10:00 am
Pacific Standard Time (San Francisco, GMT-08:00)

 

Informed consent is intended to provide patients, clinical trial participants, and others undergoing medical procedures with the information they need to make a decision about whether to undergo a specific procedure or participate in research. The process of informed consent can sometimes be very legal in nature leading to lack of clarity and misunderstanding. This webinar will explain the informed consent process, why patients should pay attention to it, and why rare disease advocates may want to get involved in the process.

Rare disease organizations play a critical role in connecting patients with researchers and the informed consent document is critically important. It outlines who will have access to research data that results from a study. Understanding the informed consent process and how to engage will help patients receive the greatest benefit.

 

Panelists:
Megan O'Boyle bio photoMegan O’Boyle

Megan’s 15-year-old daughter, Shannon has Phelan-McDermid Syndrome (PMS), an ultra rare condition. This diagnosis includes autism, intellectual disabilities, epilepsy, ADHD, lymphedema, and other medical conditions.

For the past 5 years Megan has volunteered for the PMS Foundation’s Research Support Committee. She is the Principal Investigator for the Phelan-McDermid Syndrome Data Network (PMS_DN, PCORnet) and the Phelan-McDermid Syndrome International Registry (PMSIR). She directed the biosample collection at the 2012 PMSF Family Conference, creating a biorepository of over 30 DNA and fibroblast samples.

Megan is passionate about the importance of the patient’s voice in: research, drug development, clinical trial design, development of related legislation, and quality of life decisions. She advocates for data sharing, collaborating with other advocacy groups, sharing resources, a genetics-first approach and streamlining IRB practices and policies.

Megan and her family live in Arlington, VA.

 

john-wilbanksJohn Wilbanks

John Wilbanks is the Chief Commons Officer at Sage Bionetworks. Previously, Wilbanks worked as a legislative aide to Congressman Fortney “Pete” Stark, served as the first assistant director at Harvard’s Berkman Center for Internet & Society, founded and led to acquisition the bioinformatics company Incellico, Inc., and was executive director of the Science Commons project at Creative Commons. In February 2013, in response to a We the People petition that was spearheaded by Wilbanks and signed by 65,000 people, the U.S. government announced a plan to open up taxpayer-funded research data and make it available for free. Wilbanks holds a B.A. in philosophy from Tulane University and also studied modern letters at the Sorbonne.

Moderator:
Danny_LevineDaniel Levine, Founder & Principal, Levine Media Group

Daniel Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. Since 2011, he has served as the lead editor and writer of Burrill Media’s acclaimed annual book on the biotech industry and hosts The Burrill Report’s weekly podcast. His work has appeared in The New York Times, The Industry Standard, TheStreet.com, and other national publications.

 

Register here: https://globalgenes.org/webinarinformedconsent/

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Important Recall Notice: All Auvi-Q Epinephrine Auto-Injectors

Posted on October 28, 2015 by MaryO

Sanofi US Issues Voluntary Nationwide Recall of Auvi-Q®
Due to Potential Inaccurate Dosage Delivery

 

[Press Release]

Company Contact

Karen Sutherland

Tel. : +1 908-989-0726

Email : USMediaRelations@Sanofi.com

  

 

FOR IMMEDIATE RELEASE – October 28, 2015 – Bridgewater, N.J. – Sanofi US is voluntarily recalling all Auvi-Q® (epinephrine injection, USP). The recall involves all Auvi-Q currently on the market and includes both the 0.15 mg and 0.3 mg strengths for hospitals, retailers and consumers. This includes lot number 2299596 through 3037230, which expire March 2016 through December 2016.The products have been found to potentially have inaccurate dosage delivery.
If a patient experiencing a serious allergic reaction (i.e., anaphylaxis) did not receive the intended dose, there could be significant health consequences, including death because anaphylaxis is a potentially life-threatening condition. As of October 26, 2015, Sanofi has received 26 reports of suspected device malfunctions in the US and Canada. None of these device malfunction reports have been confirmed. In these reports, patients have described symptoms of the underlying hypersensitivity reaction. No fatal outcomes have been reported among these cases.

 

Auvi-Q (epinephrine injection, USP) is used to treat life-threatening allergic reactions (anaphylaxis) in people who are at risk for or have a history of these reactions. Auvi-Q is packaged with two active devices and one trainer device in a corrugate box. Auvi-Q was distributed throughout the United States via wholesalers, pharmacies and hospitals. All Auvi-Q is being recalled.

 

auvi-q-recall

 

Sanofi US is notifying its distributors and customers who include doctors, pharmacies, wholesalers and other customers in the supply chain by letter, fax, email and phone calls and is arranging for return and reimbursement of all recalled products.

 

Customers with questions regarding this recall can go to www.Auvi-Q.com and call 1-866-726-6340 Monday through Friday 8 a.m. to 8 p.m. ET for information about how to return their Auvi-Q devices. Customers may also email cs@sanofi.com. Sanofi US will provide reimbursement for out of pocket costs incurred for the purchase of new epinephrine auto-injectors with proof of purchase.

 

Customers should immediately contact their healthcare provider (HCP) for a prescription for an alternate epinephrine auto-injector. In the event of a life-threatening allergic reaction (anaphylaxis), patients should only use their Auvi-Q device if another epinephrine auto-injector is not available, and then call 911 or local medical emergency services. Customers should contact their physician or HCP if they have experienced any problems that may be related to taking or using this drug product.


Adverse reactions or quality problems experienced with the use of this product may be reported to the FDA’s MedWatch Adverse Event Reporting program either online, by regular mail or by fax.

This recall is being conducted with the knowledge of the U.S. Food and Drug Administration.

 

Sanofi US is committed to patient safety and the quality of Auvi-Q, and will continue to work closely with customers and regulatory authorities to resolve this issue in a timely manner.

 

Important Safety Information
Auvi-Q is for immediate self (or caregiver) administration and does not take the place of emergency medical care. Seek immediate medical treatment after use. Each Auvi-Q contains a single dose of epinephrine. Auvi-Q should only be injected into your outer thigh. DO NOT INJECT INTO BUTTOCK OR INTRAVENOUSLY. If you accidentally inject Auvi-Q into any other part of your body, seek immediate medical treatment. Epinephrine should be used with caution if you have heart disease or are taking certain medicines that can cause heart-related (cardiac) symptoms.

 

If you take certain medicines, you may develop serious life-threatening side effects from epinephrine. Be sure to tell your doctor about all the medicines you take, especially medicines for asthma. Side effects may be increased in patients with certain medical conditions, or who take certain medicines. These include asthma, allergies, depression, thyroid disease, Parkinson’s disease, diabetes, high blood pressure, and heart disease.

 

The most common side effects may include increase in heart rate, stronger or irregular heartbeat, sweating, nausea and vomiting, difficulty breathing, paleness, dizziness, weakness or shakiness, headache, apprehension, nervousness, or anxiety. These side effects go away quickly, especially if you rest.

 

You are encouraged to report negative side effects of prescription drugs.

In the US, contact the FDA by visiting www.fda.gov/medwatch or call 1-800-FDA-1088.

 

Please click here for Full Prescribing Information.

 

About Sanofi US

Sanofi, an integrated global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients’ needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, consumer healthcare, emerging markets, animal health and the new Genzyme. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).

 

Sanofi is the holding company of a consolidated group of subsidiaries and operates in the United States as Sanofi US. For more information on Sanofi US, please visithttp://www.sanofi.us and http://www.news.sanofi.us/social-media or call 1-800-981-2491.

 

Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, the Group’s ability to benefit from external growth opportunities, trends in exchange rates and prevailing interest rates, the impact of cost containment policies and subsequent changes thereto, the average number of shares outstanding as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2014. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

 

From http://community.kidswithfoodallergies.org/blog/important-recall-notice-all-auvi-q-epinephrine-auto-injectors-1

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Clinical Trial for levoketoconazole

Posted on October 18, 2015 by MaryO

This trial is testing the safety and effectiveness of a new investigational drug for the treatment of Cushing’s Syndrome. Under the supervision of qualified physicians, cortisol levels and symptoms of Cushing’s Syndrome will be closely followed along with any signs of side effects.

The investigational drug (levoketoconazole) is administered by mouth in the form of tablets.

This is a phase 3 trial.

There will be up to 90 participants worldwide in this trial. This page lists U.S. sites only.

Eligibility criteria

Participants must:

be at least 18 years old
have been diagnosed with endogenous Cushing’s Syndrome by a medical professional (endogenous means that it is caused by your body producing more cortisol than it needs, not caused by the use of steroid medications)
Participants must not:

have been treated with radiation for their endogenous Cushing’s syndrome in the past 4 years
be currently using weight loss medication
have a history of drug or alcohol abuse
have been diagnosed with uncontrolled hypertension, some forms of cancer, adrenal carcinoma, Hepatitis B / C, or HIV
Note: The study doctor will ultimately determine your eligibility
Study details

The length of this study and the number of study visits will vary from patient to patient. It has approximately 13 to 27 visits to the study site spread out over one to one and a half years. This study will enroll approximately 90 participants.

A placebo isn’t being used for this trial. All study participants will receive the investigational drug.

The sponsor of this trial is Cortendo AB.

The results of this trial are intended to be published. Individual patient information will not be included.

Reasonable travel expenses may be reimbursed.

This is a global study which will be conducted in multiple countries, with several sites in the US.

This information is intended for US audiences only.

Find out if you’re eligible here.

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Hospital Staff Didn’t Give Emergency Cortisol to Teenager with Complex Special Needs

Posted on October 14, 2015 by MaryO

solu-cortef

 

A TEENAGER with complex special needs who died in hospital suffered a failure in basic medical communication, an inquest heard today.

Robin Brett, 18, of Blackmore Close, died in June 2014 in the Great Western Hospital after being admitted with chronic constipation and vomiting.

After a blood test indicated a raised white blood cell count, Robin went into cardiac arrest and died.

He had congenital adrenal hyperplasia (CAH) a metabolic disease and genetic defect of the adrenal gland and learning difficulties. He required daily care and medications.

Robin’s parents listed a string of errors they believe contributed to his death including failure to give him his regular medications, infrequent observations and the lack of regular and vital cortisol injections.

His heartbroken mother, Teresa, told the inquest in Salisbury that she had told hospital staff to give Robin an emergency dose of cortisol, which was not topped up after the recommended four hours.

She said: “I asked them to give him cortisol after I noticed he was becoming clammy and had a headache which is a sign of adrenal distress, I was just about to with the syringe in my hand when it was done and he instantly perked up.

 “But this wasn’t done again after four hours and I don’t know why.”

GWH staff nurse Hannah Porte who cared for Robin on his admission said she had concerns about his “alarmingly” high pulse rate when she did observations.

“I spoke with a doctor who assured me that because he had a pre-existing condition it wasn’t of great concern. That is our protocol and I felt comforted and reassured when they said that,” she said.

Robin was described as “rocking backwards and forwards and retching “ shortly before his cardiac arrest.

Mum Teresa broke down as she recalled her “sociable and friendly” son’s decline.

“He asked me to turn his DVD player off which was out of character in itself and he was clammy. All of a sudden he wasn’t breathing,” she said,

Registrar Fahreyer Alam, who examined Robin upon admission, said he could not provide an explanation as to why steroidal drugs were prescribed but not administered to Robin.

“They was nothing about his condition on examination which would link to adrenal crisis,” he said,

“The drugs were written on the drug chart and I cannot say why they were not given to him.”

Dr Alam said he had set an observation schedule of every two hours which he had articulated to nurses, and was not observed.

“All I can is there is an element of trust in the nurses and in a busy department we do have to relay things verbally and that is what you do,” he said.

When questioned by assistant coroner Dr Claire Balysz, Dr Alam said the effect of the seven week constipation may have put pressure on Robin’s vital organs.

“His heart and lungs were smaller than average and slightly underdeveloped. The faecal impaction made his colon stretch, it may have impacted his lungs and his heart and that is something the post mortem found,” he said.

Dr Alam and nurse Porte agreed that more was being done within the trust to improve the accuracy and accessibility of patient records, including a new observation system and the use of electronic prescribing and administration (EPMA) system.

Adapted from http://www.thisiswiltshire.co.uk/news/13843924.Robin_Brett__18__died_in_GWH_after_medics_failed_to_communicate__inquest_hears/

Filed under: adrenal, adrenal crisis, Rare Diseases | Tagged: , , , , , , , , , , , , | 2 Comments »

Cushing’s: Update on signs, symptoms and biochemical screening

Posted on October 11, 2015 by MaryO

10.1530/EJE-15-0464

  1. Lynnette Nieman⇑

+Author Affiliations


  1. L Nieman, RBMB, NIH, Bethesda, 20817-1109, United States
  1. Correspondence: Lynnette Nieman, Email: niemanl@mail.nih.gov

Abstract

Endogenous pathologic hypercortisolism, or Cushing’s syndrome, is associated with poor quality of life, morbidity and increased mortality. Early diagnosis may mitigate against this natural history of the disorder.

The clinical presentation of Cushing’s syndrome varies, in part related to the extent and duration of cortisol excess. When hypercortisolism is severe, its signs and symptoms are unmistakable. However, most of the signs and symptoms of Cushing’s syndrome are common in the general population (e.g. hypertension and weight gain) and not all are present in every patient.

In addition to classical features of glucocorticoid excess, such as proximal muscle weakness and wide purple striae, patients may present with the associated co-morbidities that are caused by hypercortisolism. These include cardiovascular disease, thromboembolic disease, psychiatric and cognitive deficits, and infections. As a result, internists and generalists must consider Cushing’s syndrome as a cause, and endocrinologists should search for and treat these co-morbidities.

Recommended tests to screen for Cushing’s syndrome include 1 mg dexamethasone suppression, urine free cortisol and late night salivary cortisol. These may be slightly elevated in patients with physiologic hypercortisolism, which should be excluded, along with exogenous glucocorticoid use. Each screening test has caveats and the choice of tests should be individualized based on each patient’s characteristics and lifestyle.

The objective of this review was to update the readership on the clinical and biochemical features of Cushing’s syndrome that are useful when evaluating patients for this diagnosis.

Read the entire manuscript at http://www.eje-online.org/content/early/2015/07/08/EJE-15-0464.full.pdf+html

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