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Data revealed a response rate of 29% for those on the therapy compared to 2% with placebo, with median platelet counts at 24 weeks of 95,000/µL in the SR group, 49,000/µL in the IR group, and 20,500/µL in the non-responders.

This morning, the FDA granted Orphan Drug Designation to KL1333 for the treatment of inherited mitochondrial respiratory chain diseases (MRCD).

Teens who self-reported inattention, hyperactivity/impulsivity, and conduct disorder also reported higher risky driving behaviors.

A survey recently conducted among adults with hereditary spastic paraparesis showed that constipation, alternate constipation and diarrhea, fecal incontinence, and voiding dysfunction are among the considerable problems faced by the patient population.

Clostridium difficile could be mutating to transfer from pets to humans.

Patients using statins had a 4.3% infection rate compared with a 7.8% rate for those not taking statins, concluding that statins may be associated with a 20% risk reduction.

Rigel announced that the U.S. FDA approved fostamatinib disodium hexahydrate (Tavalisse) tablets for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia.

Burosumab is the first FDA approved treatment for this rare, hereditary disorder that affects 3000 children and 12,000 adults in the US.

FDA grants Breakthrough Therapy Designation to emicizumab-kxwh (Hemlibra) for people with hemophilia A without factor VIII inhibitors.

The disparity of fast food swamps significantly affecting pediatric patients with T1D but not those with T2D came as a surprise to researchers.

Burosumab (Crysvita) was approved this morning by the U.S. FDA to treat adults and children ages 1 year and older with x-linked hypophosphatemia (XLH), a rare genetic variation of rickets.

This morning, Shire announced the expanded U.S. FDA approval of Vonvendi, a recombinant von Willebrand factor, for the treatment of bleeding in adults with von Willebrand disease in surgical settings.

Can the Epstein-Barr virus cause autoimmune illnesses?

SGLT-2 inhibitors and GLP-1 agonists were associated with 1% and 0.6% reductions in all-cause mortality, respectively.

Data presented at the 2018 American Association for Cancer Research Annual Meeting show that a new combination could provide long-term, systemic responses in patients with advanced melanoma.

Treatment now offers an individualized approach to control bleeding in appropriate patients undergoing surgery.

The therapy, when used prophylactically either weekly or biweekly, showed a statistically significant reduction in treated bleeds in comparison to no prophylaxis.

This morning, the U.S. FDA provided full approval to Boehringer Ingelheim for idarucizumab (Praxbind), its specific reversal agent for dabigatran etexilate mesylate (Pradaxa).

In 90 patients that received idarucizumab in the RE-VERSE AD phase 3 trial, the median maximum percentage reversal was 100%.

An Alzheimer’s diagnosis may cause fear, but the broad spectrum of cognitive impairment means some physicians can continue to practice despite the disease.

Current research shows that positive parenting strategies are linked with improved social-emotional development, but gaps in analysis persist.

The likelihood that a patient would receive a new opioid prescription was higher for at-risk patients and remained true even after adjusting for demographic characteristics, comorbidities, and diagnoses associated with pain.

The therapy, FX1a inhibitor BMS-986177, will advance to phase 2 clinical trials for wider indications.

FDA approves the development of VAR 200 for the treatment of focal segmental glomerulosclerosis (FSGS) in adults.

FDA exercises enforcement discretion regarding its ExeGen ATM MiniSwine model, clearing the genetically engineered (GE) to model ataxia telangiectasia (AT) for commercial use as a research tool.

Irritability was associated with widespread neural activation or increased activity in the insula, caudate, dorsolateral and ventrolateral prefrontal cortex, and inferior parietal lobule.

This morning, Galapagos announced the design of its global Phase 3 program of GLPG1690 in patients with idiopathic pulmonary fibrosis (IPF).

Transgender/gender-nonconforming youth may present with mental health conditions that require immediate evaluation and implementation gender identity support measures.

The angle of the superior cerebellar artery outlet can be useful for monitoring the progression of basilar artery dolichoectasia in patients with Pompe, according to results in the Orphanet Journal of Rare Diseases.

Mobile apps have a high potential for improving treatment adherence, but rigorous testing is still needed to determine efficacy.