Zafgen shelves another drug; chief medical officer exits
The bad news keeps coming for Boston biotech Zafgen Inc., which revealed Monday that it is shelving another drug due to safety concerns. Zafgen (Nasdaq: ZFGN) has hit multiple dead ends while attempting to treat metabolic diseases such as Type 2 diabetes and Prader-Willi Syndrome by inhibiting an enzyme known as MetAP2. Testing on the company's lead drug for Type 2 diabetes, ZGN-1061, remains on a clinical hold in the U.S. nearly six months after the FDA voiced concerns about a potential cardiovascular… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - March 12, 2019 Category: Health Management Authors: Allison DeAngelis Source Type: news

Guanfacine extended release for the reduction of aggression, attention-deficit/hyperactivity disorder symptoms, and self-injurious behavior in Prader-Willi syndrome-a retrospective cohort study - Singh D, Wakimoto Y, Filangieri C, Pinkhasov A, Angulo M.
OBJECTIVE: To examine the role of Guanfacine Extended Release (GXR) in the management of behavioral disturbances in patients with Prader-Willi Syndrome (PWS). METHODS: Twenty from a total of 27 individuals with genetically confirmed PWS, 6-26 years... (Source: SafetyLit)
Source: SafetyLit - February 11, 2019 Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news

What is Genomic Imprinting?
Discussion Silver-Russell syndrome (SRS) is a rare genetic syndrome first characterized by Silver in 1953 and Russell in 1954. Patients with SRS have characteristic growth patterns and clinical findings, although within an individual patient there are phenotypical differences. Patients are born small-for-gestational age (SGA) but have a relative macrocephaly. There is postnatal growth failure and difficulty feeding, with a very low body mass index. Body asymmetry (e.g. hemihypertrophy) and facial features (i.e. protruding forehead, triangular facies, micrognathia, dental anomalies, downturned mouth corners, and ear anomali...
Source: PediatricEducation.org - November 26, 2018 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Mother, 36, ignores her daughter’s screams for food as rare condition leaves her constantly hungry
Megan Fitzgerald, seven, from Hull, has Prader-Willi Syndrome. Her mother, Tracy Fitzgerald, 36, says she looks like a 'horrible mother' in public, and can't even speak about food in front of her child. (Source: the Mail online | Health)
Source: the Mail online | Health - November 22, 2018 Category: Consumer Health News Source Type: news

Mother, 36, ignores her daughter ’s screams for food as rare condition leaves her constantly hungry
Megan Fitzgerald, seven, from Hull, has Prader-Willi Syndrome. Her mother, Tracy Fitzgerald, 36, says she looks like a 'horrible mother' in public, and can't even speak about food in front of her child. (Source: the Mail online | Health)
Source: the Mail online | Health - November 22, 2018 Category: Consumer Health News Source Type: news

Anna Hankins who suffers from Prader-Willi Syndrome wins beauty pageant
Anna Hankins, from Louisville, Mississippi, who was diagnosed with Prader-Willi Syndrome at two years old, developed an insatiable appetite as a toddler, which caused her to steal food off plates. (Source: the Mail online | Health)
Source: the Mail online | Health - March 26, 2018 Category: Consumer Health News Source Type: news

Teenage girl, 15, suffers from a rare condition that makes  her so hungry she rifles through bins
Anna Hankins, from Louisville, Mississippi, who was diagnosed with Prader-Willi Syndrome at two years old, developed an insatiable appetite as a toddler, which caused her to steal food off plates. (Source: the Mail online | Health)
Source: the Mail online | Health - March 26, 2018 Category: Consumer Health News Source Type: news

An enzyme deficiency contributes to disease symptoms in Prader-Willi syndrome
Researchers discovered that a deficiency of the enzyme prohormone convertase 1, contributes to disease symptoms in Prader-Willi Syndrome, a genetic disorder. (Source: NIDDK News)
Source: NIDDK News - March 15, 2018 Category: Endocrinology Source Type: news

Letting silenced genes speak
(University of Connecticut) Stem cell researchers at UConn Health have reversed Prader-Willi syndrome in brain cells growing in the lab. The discovery provides clues that could lead to a treatment for Prader-Willi, a genetic disorder that silences genes necessary for normal brain function. Prader-Willi occurs in about one out of every 15,000 births, and is the most common genetic cause of life-threatening childhood obesity. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 25, 2018 Category: International Medicine & Public Health Source Type: news

FDA Grants INSYS Therapeutics ‘Fast Track’ Designation for Cannabidiol (CBD) Oral Solution as Investigational Treatment for Prader-Willi Syndrome
PHOENIX, Dec. 26, 2017 -- (Healthcare Sales & Marketing Network) -- INSYS Therapeutics, Inc. (NASDAQ:INSY), announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company’s cannabidiol (CBD) oral solution... Biopharmaceuticals, FDA INSYS Therapeutics, cannabidiol, Prader-Willi Syndrome (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - December 26, 2017 Category: Pharmaceuticals Source Type: news

Seven-year-old who weighs 19 stone has weight-loss surgery
Muhammad Yousuf from Kasur in Punjab, Pakistan, weighs four times more than an average child of his age. He is thought to suffer from Prader-Willi syndrome, which causes an insatiable appetite. (Source: the Mail online | Health)
Source: the Mail online | Health - December 6, 2017 Category: Consumer Health News Source Type: news

Pakistani boy who weighs 19 stone has weight-loss surgery
Muhammad Yousuf, seven, from Kasur in Pakistan, weighs four times more than an average child of his age. He is thought to suffer from Prader-Willi syndrome, which causes an insatiable appetite. (Source: the Mail online | Health)
Source: the Mail online | Health - December 6, 2017 Category: Consumer Health News Source Type: news

Boy with Prader-Willi syndrome means never feels full
Frankie Udall, from Billericay, Essex, suffers from Prader-Willi syndrome, an incurable disease that can make him scream and lash out due to his hunger. (Source: the Mail online | Health)
Source: the Mail online | Health - November 13, 2017 Category: Consumer Health News Source Type: news

Is THIS Mexican boy the world's fattest baby?
Luis Manuel, 10 months, from Tecoman in the western Mexican state of Colima, tips the scales at a mammoth 4st 10lbs (30kg). Doctors fear he has the incurable Prader-Willi syndrome. (Source: the Mail online | Health)
Source: the Mail online | Health - October 20, 2017 Category: Consumer Health News Source Type: news

The little girl who is always hungry
Six-year-old Megan has the rare Prader-Willi syndrome, which means she never feels full. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - October 13, 2017 Category: Consumer Health News Source Type: news

The boy who used to eat enough to feed 10 PEOPLE
Mahesh Bhubathi, 15, from Anantapur district in southern India, suffers from Prader-Willi syndrome - a genetic disorder which often leaves children constantly hungry. (Source: the Mail online | Health)
Source: the Mail online | Health - April 6, 2017 Category: Consumer Health News Source Type: news

Oxytocin boosts social skills in autism-related syndrome
New clinical trial, published inPediatrics, finds that one week of treatment with oxytocin improves social skills and feeding in infants with Prader-Willi syndrome.Spectrum News (Source: Society for Endocrinology)
Source: Society for Endocrinology - March 17, 2017 Category: Endocrinology Source Type: news

GH Treatment Appears to Boost Height in Prader-Willi Patients GH Treatment Appears to Boost Height in Prader-Willi Patients
Growth-hormone treatment appears to improve linear growth in children with Prader-Willi syndrome (PWS), a new analysis of registry data confirms.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - March 3, 2017 Category: Consumer Health News Tags: Medscape Today News Source Type: news

Experimental therapy for Prader-Willi syndrome shows promise in mice
Drugs capable of activating silenced genes improve survival and growth outcomes in a mouse model of Prader-Willi syndrome (PWS), a rare and incurable childhood disease. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - December 27, 2016 Category: Science Source Type: news

Experimental therapy for Prader-Willi syndrome shows promise in mice
(NIH/Eunice Kennedy Shriver National Institute of Child Health and Human Development) Drugs capable of activating silenced genes improve survival and growth outcomes in a mouse model of Prader-Willi syndrome (PWS), a rare and incurable childhood disease, according to a study funded by the National Institutes of Health (NIH). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 26, 2016 Category: Global & Universal Source Type: news

Potential drug appears to ease effects of Prader-Willi syndrome
(Duke University Medical Center) Duke Health researchers have identified a drug-like small molecule that, in animal experiments, appears to be an effective treatment for a genetic disorder called Prader-Willi syndrome. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 26, 2016 Category: Global & Universal Source Type: news

Rare obesity syndrome therapeutic target identified
An enzyme deficiency in the brain is linked to Prader-Willi syndrome, a genetic condition that causes extreme hunger and severe obesity beginning in childhood, scientists have discovered. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - December 13, 2016 Category: Science Source Type: news

23 stone 11-year-old set on FIRE and undergoes cupping treatment to lose weight  in China
Li Hang, from China's Harbin city, suffers from Prader-Willi Syndrome, a rare genetic disorder. The child is going through traditional Chinese medicine treatment to lose weight. (Source: the Mail online | Health)
Source: the Mail online | Health - November 25, 2016 Category: Consumer Health News Source Type: news

The four-year-old who screams when she isn't fed on time
Hailey Gossage-Paul, from St Louis, Missouri, is given her breakfast at 7.30am precisely, lunch at midday on the dot and dinner not a second later than 4.35pm to cope with her Prader-Willi syndrome. (Source: the Mail online | Health)
Source: the Mail online | Health - November 17, 2016 Category: Consumer Health News Source Type: news

Aripiprazole in the treatment of obsessive compulsive disorder and aggressive behaviors in a child with Prader Willi syndrome: a case report - Akca OF, Yilmaz S.
[Abstract unavailable] Language: en... (Source: SafetyLit)
Source: SafetyLit - August 20, 2016 Category: Global & Universal Tags: Age: Infants and Children Source Type: news

Twin Brothers Battle Rare Condition That Causes Never-Ending Hunger
Raising twin boys is a challenge for any single mom, but it’s especially difficult for one particular mom in New Haven, Connecticut. Dianna Schatzlein-Ahern’s two youngest sons, Stevie and Eddie Ahern, are 11. They both suffer from Prader-Willi syndrome, a genetic condition that makes them insatiably hungry no matter how much they eat. “I have to keep everything locked away so they don’t get up in the middle of the night and have a feast,” Schatzlein-Ahern told Barcroft TV. “They can never feel full, so could potentially eat themselves to death.” For health reasons, she a...
Source: Science - The Huffington Post - July 28, 2016 Category: Science Source Type: news

Alizé Pharma to report Phase II trial results of AZP-531 to treat PWS
French-based biopharmaceutical company Alizé Pharma will report results of its Phase II trial of AZP-531 to treat patients with Prader-Willi (PWS) syndrome. (Source: Drug Development Technology)
Source: Drug Development Technology - July 20, 2016 Category: Pharmaceuticals Source Type: news

Aliz é Pharma to report Phase II trial results of AZP-531 to treat PWS
French-based biopharmaceutical company Aliz é Pharma will report results of its Phase II trial of AZP-531 to treat patients with Prader-Willi (PWS) syndrome. (Source: Drug Development Technology)
Source: Drug Development Technology - July 20, 2016 Category: Pharmaceuticals Source Type: news

New insights uncovered into Prader-Willi syndrome
(Children's Hospital Los Angeles) A study published in the journal Human Molecular Genetics by researchers at Children's Hospital Los Angeles provides novel insights into the brain mechanisms underlying the insatiable hunger and subsequent obesity in patients with Prader-Willi syndrome. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 14, 2016 Category: Global & Universal Source Type: news

Unexpected function of small nucleolar RNAs explains cause of some diseases
Scientists have discovered unexpected functions of small nucleolar RNAs (snoRNAs) that explain the cause of some diseases. They found that snoRNAs not only modify ribosomes, but can also regulate alternative splicing. Through this second function, they regulate protein function and inhibit the generation of wrong protein variants. Thus, upon the loss of snoRNAs the formation of wrong protein variants can no longer be prevented, leading to diseases like Prader-Willi syndrome and several cancers, report scientists. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - June 7, 2016 Category: Science Source Type: news

Scientists find new roles for old RNAs
Scientists have discovered unexpected functions of small nucleolar RNAs (snoRNAs) that explain the cause of some diseases. The loss of small nucleolar RNAs is associated with a number of diseases, including Prader-Willi syndrome and several forms of cancers; and genetic duplications of some snoRNAs could play a role in autism. However, it is not clear how the change in snoRNA expression could lead to these diseases. Using RNA sequencing and molecular biology techniques, the researchers found that snoRNAs not only modify ribosomes, but can also regulate alternative splicing. Through this second function, they regulate prote...
Source: ScienceDaily Headlines - June 6, 2016 Category: Science Source Type: news

Unexpected function of small nucleolar RNAs explains cause of some diseases
(The Hebrew University of Jerusalem) Scientists have discovered unexpected functions of small nucleolar RNAs (snoRNAs) that explain the cause of some diseases. They found that snoRNAs not only modify ribosomes, but can also regulate alternative splicing. Through this second function, they regulate protein function and inhibit the generation of wrong protein variants. Thus, upon the loss of snoRNAs the formation of wrong protein variants can no longer be prevented, leading to diseases like Prader-Willi syndrome and several cancers. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - June 6, 2016 Category: Cancer & Oncology Source Type: news

Beloranib reduces weight in Prader-Willi syndrome, but concerns remain
BOSTON – Prader-Willi syndrome patients lost more than 4% of their body weight over a 26-week period with the investigative antiobesity drug beloranib, compared with a similar gain in patients taking... (Source: Clinical Endocrinology News)
Source: Clinical Endocrinology News - May 17, 2016 Category: Endocrinology Source Type: news

Beloranib reduces weight in Prader-Willi syndrome, but concerns remain
BOSTON – Prader-Willi syndrome patients lost more than 4% of their body weight over a 26-week period with the investigative antiobesity drug beloranib, compared with a similar gain in patients taking... (Source: Family Practice News)
Source: Family Practice News - May 17, 2016 Category: Primary Care Source Type: news

Zafgen and the fight against obesity
Presented at the annual conference of the Endocrine Society, a novel drug may offer hope in Prader-Willi Syndrome and obesity. Medscape (Source: Society for Endocrinology)
Source: Society for Endocrinology - April 7, 2016 Category: Endocrinology Source Type: news

Novel Drug May Offer Hope in Prader-Willi Syndrome, ObesityNovel Drug May Offer Hope in Prader-Willi Syndrome, Obesity
Beloranib reduces weight and hyperphagia in Prader-Willi syndrome and may also hold promise for other severe obesity conditions, but first the company must prove that it doesn't raise thrombotic risk. Medscape Medical News (Source: Medscape Diabetes Headlines)
Source: Medscape Diabetes Headlines - April 6, 2016 Category: Endocrinology Tags: Diabetes & Endocrinology News Source Type: news

Rare disorder causes constant hunger
Peggy Ickenroth photographed Suzanne, a 12-year-old with Prader-Willi syndrome. The genetic disorder's most prominent symptom is an insatiable appetite -- you never feel full. (Source: CNN.com - Health)
Source: CNN.com - Health - March 14, 2016 Category: Consumer Health News Source Type: news

Zafgen's Phase III trial of obesity drug Beloranib shows efficacy
Biopharmaceutical company Zafgen announced that the Phase III trial of its experimental obesity drug Beloranib in Prader-Willi syndrome (PWS) has achieved co-primary efficacy endpoints. (Source: Drug Development Technology)
Source: Drug Development Technology - January 21, 2016 Category: Pharmaceuticals Source Type: news

What Types of Hand Stereotypies are Associated with Rett Syndrome?
Discussion Lady MacBeth and patients with Rett Syndrome (RS) are obviously not alike other than in making similar hand motions. In Shakespeare’s MacBeth (5.1.25-30), Lady MacBeth was driven into madness because of her murderous guilt and tries to wash the blood off her hands as she sleepwalks. Doctor. What is it she does now? Look, how she rubs/ her hands. Gentlewoman. It is an accustomed action with her, to seem thus/ washing her hands. I have known her continue in this/ a quarter of an hour. In 1954, Dr. Andreas Rett observed 2 girls who were also making repetitive handwashing motions and later published a case...
Source: PediatricEducation.org - January 11, 2016 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Second patient dies during clinical trial for obesity drug
Stephen FellerWASHINGTON, Dec. 2 (UPI) -- A second patient has died in a clinical trial for the drug beloranib, which is being tested for use with a rare obesity disorder called Prader-Willi syndrome. (Source: Health News - UPI.com)
Source: Health News - UPI.com - December 2, 2015 Category: Consumer Health News Source Type: news

Aliya Saleem is one of the heaviest girls in the world 'due to genetic condition'
Aliya Saleem, 18 months, of Jharkhand, eastern India, may be suffering from Prader-Willi syndrome, a rare disorder characterised by obsessive eating and learning difficulties. (Source: the Mail online | Health)
Source: the Mail online | Health - December 2, 2015 Category: Consumer Health News Source Type: news

Patient dies during Zafgen's obesity drug trial
(Reuters) - Zafgen Inc said a patient died during a late-stage trial testing the company's drug to treat Prader-Willi syndrome, a rare genetic disorder that leads to obesity. (Source: Reuters: Health)
Source: Reuters: Health - October 19, 2015 Category: Consumer Health News Tags: healthNews Source Type: news

Boston biotech Zafgen shares down after patient dies in obesity drug trial
Shares of Zafgen plunged today after the small biotech firm said a patient in a late-stage trial of its experimental drug to treat obesity has died. In a statement, the company said that "Zafgen (Nasdaq: ZFGN) recently learned of a patient death which occurred in the company’s ongoing double-blind, randomized, placebo-controlled Phase 3 best PWS study of beloranib in Prader-Willi Syndrome, a rare genetic disorder with a high rate of mortality linked to obesity and its co-morbidities. The cause… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 14, 2015 Category: Biotechnology Authors: Don Seiffert Source Type: news

Weight loss surgery offers new hope to children, adolescents with Prader-Willi Syndrome
Obesity is a leading cause of complications and death in children suffering from Prader-Willi syndrome (PWS), yet there are few effective treatment options for these patients. In a new study, researchers found that bariatric surgery, specifically laparoscopic sleeve gastrectomy (LSG), resulted in substantial weight loss with no apparent adverse effect on growth in a small group of severely overweight patients with PWS. PWS is a rare genetic condition that causes a wide range of problems including a constant desire to consume food, which is driven by a permanent feeling of hunger. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - September 28, 2015 Category: Science Source Type: news

Weight loss surgery offers new hope to children and adolescents with Prader-Willi Syndrome
(Elsevier Health Sciences) Obesity is a leading cause of complications and death in children suffering from Prader-Willi syndrome (PWS), yet there are few effective treatment options for these patients. In a new study researchers found that bariatric surgery, specifically laparoscopic sleeve gastrectomy (LSG), resulted in substantial weight loss with no apparent adverse effect on growth in a small group of severely overweight patients with PWS. While bariatric surgery is considered controversial for PWS, the research team is encouraged by their positive results. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 28, 2015 Category: Global & Universal Source Type: news

Morbidly obese woman with Prader-Willi syndrome ate herself to death
Kirsty Derry, 23, of Heath Hayes, Staffordshire, died from heart failure after putting on 7st in nine months after moving into an assisted living care home Victoria Mews in Stone, Staffordshire. (Source: the Mail online | Health)
Source: the Mail online | Health - July 13, 2015 Category: Consumer Health News Source Type: news