Metabolic Disorders

Kalydeco Approved to Treat Cystic Fibrosis in Children 12 to <24 Months

By August 17, 2018

The approval was supported by data from ARRIVAL (N=25), an ongoing Phase 3 open-label safety study in children with CF aged 12 to <24 months with 1 of 10 CFTR gene mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H).

Low-Energy Diet Induces Different Effects in Men, Women

August 15, 2018

Men had larger reductions in metabolic syndrome Z-score, C-peptide, fat mass, heart rate

Galafold Approved for the Treatment of Adults With Fabry Disease

By August 13, 2018

The indication was approved under an accelerated approval pathway based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate.

First-in-Class Therapy Approved for Polyneuropathy Caused by hATTR

By August 10, 2018

The mechanism of siRNAs consists of silencing a portion of RNA involved in causing the disease.

FDA Grants Orphan Drug Status to Angelman Syndrome Treatment

By August 10, 2018

Delay or lack of speech, seizures, and walking/balance disorders are some of the symptoms associated with AS.

AHA: A Diverse Diet May Not Promote a Healthy Weight

August 10, 2018

The authors report that current data do not support greater dietary diversity as an effective strategy to promote healthy eating patterns and healthy body weight.

Orkambi Approved to Treat Younger Cystic Fibrosis Patients

By August 08, 2018

The approval was based on data from a 24-week, open-label, Phase 3 study in patients aged 2 to 5 years.

PCOS and Overweight/Obesity: Drug Therapies for Weight Reduction Compared

By August 06, 2018

Researchers identified 23 randomized-controlled trials that included patients with PCOS and overweight/obesity (N=941) and compared the effectiveness of various pharmacological interventions (i.e., metformin, inositol, liraglutide, orlistat) on weight reduction.

Obesity Treatments

August 03, 2018

A comparative list of medications by pharmacological class for the treatment of obesity.

Treatment for Prader-Willi Syndrome Gets Fast Track Designation

By July 31, 2018

Five Phase 1 and three Phase 2 clinical trials of DCCR have been completed, with one of the Phase 2 trials involving PWS patients.

Results From Long-Term Cardiovascular Safety Study of Lorcaserin Announced

By July 19, 2018

Approximately 12,000 patients took part in the CAMELLIA-TIMI 61 trial in which participants were randomized to either lorcaserin HCl 10mg twice daily or placebo.

Parents Urged to 'Look Before You Lock' Cars

July 10, 2018

"These children don't have to die," KidsAndCars.org founder and president Janette Fennell said in a statement. "Parents and caregivers have the power to make sure that this doesn't happen to them."

Occupational Exposure Limits for Heat Stress Often Exceeded: CDC

July 09, 2018

For the same 25 cases, a Heat Index screening threshold of 85 degrees Fahrenheit could identify potentially hazardous levels of workplace environmental heat when WBGT was unavailable. Six fatalities occurred when the Heat Index was <91 degrees Fahrenheit.

Menkes Disease Treatment Granted Fast Track Status by FDA

By July 02, 2018

CUTX-101, a subcutaneous injectable formulation of copper histidinate, is designed to supplement blood and brain copper levels in patients with Menkes disease.

Azithromycin Cuts Pulmonary Exacerbation in CF With Early <i>Pa</i>

June 28, 2018

Risk of pulmonary exacerbation down for children with cystic fibrosis, early Pseudomonas aeruginosa

Many Drugs Made Available via FDA Expanded Access Programs

June 20, 2018

Medicines to treat cancer (50%); metabolic, endocrine, and genetic diseases (17.4%); and infectious diseases (5.2%) were most common in these programs. Ninety-two FDA-approved drugs and biologics with associated expanded access programs initiated prior to FDA approval between September 1996 and June 2017.

Adult Obesity Prevalence Varies With Level of Urbanization

June 20, 2018

Age-adjusted prevalence of obesity was increased for men living in medium or small metropolitan statistical areas (MSAs) versus those living in large MSAs (42.4 vs 31.8%); the difference for men living in large MSAs vs non-MSAs was not statistically significant.

Tattoo-Linked Complications ID'd in Immunosuppressed Female

June 19, 2018

This case details complications related to tattooing in a 31-year-old woman with a history of cystic fibrosis who had undergone bilateral lung transplantation in 2009 and was on long-term immunosuppressive therapy.

Gene Therapy for Mucopolysaccharidosis Type I Gets Fast Track Designation

By June 15, 2018

Current treatments for MPS 1 include bone marrow transplant and enzyme replacement with a recombinant form of human IDUA administered intravenously.

FDA Approves Updated Labeling for Two Obesity Treatments

By June 04, 2018

Specifically, the new labeling includes updated data on the global rate of occurrence related to these adverse events.

CVD Risk Up Even in Metabolically Healthy Obese Women

May 31, 2018

Compared to women with stable healthy normal-weight, women who maintained metabolically healthy obesity during follow-up were still at elevated cardiovascular risk (hazard ratio, 1.57); this risk was lower than for initially metabolically healthy women who converted to an unhealthy phenotype (hazard ratios, 1.90 and 2.74 for normal-weight and obesity, respectively).

First Enzyme Substitution Therapy Approved for Phenylketonuria in Adults

By May 25, 2018

Palynziq is a PEGylated phenylalanine ammonia lyase enzyme that converts phenylalanine to ammonia and trans-cinnamic acid.

Antidepressant Prescribing Linked to Lasting Weight Gain

May 25, 2018

During 1,836,452 person-years of follow-up, the incidence of new episodes of ≥5% weight gain was 8.1 and 11.2 per 100 person-years in participants not prescribed and prescribed antidepressants, respectively (adjusted rate ratio, 1.21).

Lenti-D Gets Breakthrough Tx Designation for Cerebral Adrenoleukodystrophy

By May 24, 2018

This investigational lentiviral-based gene therapy is currently being studied in the Phase 2/3 Starbeam Study, a 2-year study assessing the safety and efficacy of Lenti-D in boys ≤17 years old with CALD.

Primary Care Providers Have Mixed Views on Genetic Tests

May 14, 2018

They find tests for genetic risk potentially useful, but lack confidence in interpreting results

FDA Committee in Favor of Waylivra for Rare Metabolic Lipid Disorder

By May 11, 2018

Antisense technology refers to the use of synthetic nucleic acid sequences to interrupt specific protein production by targeting the corresponding messenger RNA that encodes that protein.

Lesbian, Bi Women More Likely to Develop T2DM at Younger Age

May 09, 2018

Higher BMI in lesbian and bisexual women is an important contributor to the greater level of type 2 diabetes among this population, the authors found.

Improved research methods needed to determine effectiveness of anti-obesity public health initiatives

May 02, 2018

Researchers found that amongst nearly 300 studies reporting the impact of policies, programs, or changes in built environment on controlling and preventing obesity, most natural experiments had high risk of bias and poor handling of dropouts and withdrawals.

Severe Obesity Tied to Faster Progression to Disability With RA

May 01, 2018

The researchers found that at baseline, severely obese patients had higher disability scores versus overweight patients in both Forward and VARA.

Review: Need to Strengthen Natural Experiments in Obesity

May 01, 2018

In terms of study design, guidance, documentation, and communication about methods used should be improved. Cross-cutting recommendations are provided for improving the rigor of natural experiments in obesity.