Metabolic Disorders

Pompe Disease Treatment Granted Orphan Drug Designation

By September 25, 2017

ATB200 is a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures while AT2221 is a pharmacological chaperone.

Increased Med Use Over Time Seen in Older Patients of All BMI Classes

By September 22, 2017

Researchers aimed to determine whether the rise in prescription drug use over time varied by age and obesity status.

Safety, Effectiveness of Cryolipolysis for Noninvasive Reduction of Fat

September 22, 2017

The researchers found that the mean reduction measured by skin fold caliper was 33% (3.2 mm; P=0.05), and by magnetic resonance imaging, the mean reduction was 1.78mm.

Long-Term Outcomes of Roux-en-Y Gastric Bypass

September 21, 2017

The researchers found that the adjusted mean changes from baseline in body weight in the surgery group were −45.0, −36.3, and −35.0 kg at 2, 6, and 12 years, respectively.

Weight May Play Role in Selecting Treatment Strategy for Breast CA Patients

September 20, 2017

BMI was positively correlated with ER+/PR+ and ER−/PR− subtype risks, with a significant association only seen for ER+/PR+ subtypes.

Investigational Skin Patch Delivers Fat-Burning Drug to Targeted Areas

By September 15, 2017

The patches mechanism of action turns white adipose tissue into brown adipose tissue, white tissue is known to store fat while brown tissue is known to burn fat to produce heat.

Stress, Sleep, Obesity Connection Explored in New Study

September 05, 2017

Stress linked to impaired sleep, increased emotional rewarding of palatable foods, leading to obesity

Reduced Dosing Option Approved for Orfadin in HT-1

By September 05, 2017

The approval of the once-daily dosing option was based on a clinical study that compared a 4-week once-daily regimen to a 4-week twice daily regimen.

Tezacaftor/Ivacaftor Gets Priority Review for Cystic Fibrosis

By August 25, 2017

The FDA has granted Priority Review to the New Drug Application (NDA) of tezacaftor/ivacaftor (Vertex) for the treatment of patients ≥12yrs old with cystic fibrosis (CF) who have two copies of the F508del mutation or one F508del mutation and one residual function mutation.

Exendin Assessed for Post-Bariatric Complications

August 11, 2017

The postprandial glucose nadir was increased by 66%, peak insulin was reduced by 57%, and neuroglycopenic symptoms were reduced by 80%, on average.

FDA: Deaths Following Intragastric Balloon Placement for Obesity

By August 10, 2017

Two additional reports of death within the same time period may possibly be related to balloon placement as well.

Significant Associations Found Between Religiosity and Body Weight

August 07, 2017

The researchers found that for religious affiliation there were significant associations with body weight in both cross-sectional and longitudinal studies.

Treatment Shows Efficacy in Binge Eating Disorder

August 04, 2017

In both studies, more participants on LDX versus placebo were categorized as improved on the Clinical Global Impressions-Improvement starting at week one.

New Treatment Option for Hereditary Tyrosinemia Type 1 Approved

By August 01, 2017

The safety and efficacy of Nityr have been established based on studies of another oral formulation of nitisinone in patients with HT-1.

FDA Approves Kalydeco to Treat More Patients with Cystic Fibrosis

By August 01, 2017

Today's FDA approval follows the Agency's recent approval for 23 other residual function mutations based on in vitro data.

Serum γ-Glutamyltransferase Levels May ID MetS in Children

July 28, 2017

After adjustment for age, household income, and residential area, the multivariable-adjusted odds ratios for MetS for the upper stratum were 5.79 and 6.20 in boys and girls, respectively.

'Overfat' Pandemic Grows Even as Obesity Rates Level Off

July 25, 2017

Traditional methods of measuring weight or body mass index are ineffective in assessing whether a person is overfat, the study authors said.

Weight Gain During Adulthood Ups Risk of Major Chronic Diseases

July 20, 2017

As little as 11 pounds gained from early to middle adulthood tied to significantly elevated risk

Aspirin-Induced Platelet Inhibition May Be More Potent Post-Bariatric Sx

July 20, 2017

The researchers found that off-treatment ARU was significantly reduced post-bariatric surgery versus pre-surgery levels (602±59 versus 531±78; P=0.035).

Correlation Between Weight and Heart Failure Explored

July 20, 2017

The investigators found that people who increased their weight by as little as 5% were more likely to have thickening and enlargement of the left ventricle.

First-in-Class Drug for Primary Hyperoxaluria Gets Orphan Drug Designation

By July 13, 2017

The Orphan Drug designation was supported by data from preliminary studies showing that ALLN-177 significantly decreased urinary and plasma oxalate in several rodent and porcine models.

Sugary Drink Consumption During Pregnancy Tied to Adiposity in Offspring

July 11, 2017

The team found that higher sugar-sweetened beverage intake during the second trimester of pregnancy was associated with greater adiposity in mid-childhood.

SOBI003 Gains Orphan Drug Status for Rare Metabolic Disorder

By July 05, 2017

According to the Company, SOBI003 is currently in the late pre-clinical phase and its first clinical trial is expected to commence in 2018.

Panhematin Available in New Dosage Strength

By July 05, 2017

The new dosage strength better meets the required dose of most patients with acute intermittent porphyria.

Biomarin Submits BLA for New Phenylketonuria Therapy

By July 05, 2017

The investigational drug is intended to substitute the deficient PAH enzyme with a PEGylated phenylalanine lyase enzyme to break down Phe.

Impact of E-Cigarettes on Weight, Metabolic Parameters Examined

July 05, 2017

Even without nicotine exposure, similar delirious effects for e-cigarettes, traditional tobacco cigarettes

Flaxseed Consumption Tied to Weight Reduction

June 30, 2017

Significant reduction in body weight, BMI, waist circumference following flaxseed consumption

Obesity May Increase Influenza Risk Among Vaccinated Patients

By June 27, 2017

The researchers assessed the relative risk for influenza and ILI on the basis of body mass index and determined seroconversion and seroprotection rates using pre-vaccination and 26-35 day post-vaccination serum samples.

Novel Synthetic Bio Drug for Hyperammonemia Granted Fast Track

By June 26, 2017

Synthetic Biotic medicines utilize synthetic biology to reprogram probiotic bacteria to perform critical functions that compensate for those missing or damaged due to a particular disease.

USPSTF Updates Obesity Screening Recommendations for Children

June 21, 2017

B recommendation supports screening for children and adolescents aged 6 and older