Matt Hoffman

Part 2 of the pivotal FIREFISH clinical trial met its primary end point in infants aged 1 to 7 months with Type 1 spinal muscular atrophy, with statistically significant and medically meaningful motor milestone improvements.

The Headaterm TENS device was shown to decrease pain at an 11.8% better rate than many acute migraine medications used in the emergency department in randomized trials, with data suggesting it is both effective and fast-acting.

The novel oxybate agent has been submitted as an investigational treatment for cataplexy and excessive daytime sleepiness in those ages 7 years and older with narcolepsy.

The medical director of the Comprehensive Epilepsy Clinic at Nicklaus Children’s Hospital shared his insight into the potential of using an SCN1A-targeted adeno-associated viral vector-based gene therapy in epilepsy.

Those who have strokes with favorable imaging profiles have a higher likelihood of receiving endovascular thrombectomy as well as achieving high functional independence rates after 90 days.

As was the case for siponimod’s FDA approval in March 2019, this regulatory decision by the European Commission for the selective sphingosine-1-phosphate receptor modulator was based on the phase 3 EXPAND trial.

Use of rimabotulinumtoxinB (Myobloc; US WorldMeds) result in statistically significant reductions in sialorrhea compared to placebo with both 2500 U and 3500 U doses.

After first-step data suggested that one small molecule CGRP receptor antagonist and a monoclonal antibody against CGRP were successful and safe together in migraine. One of the study authors, Kathleen Mullin, MD, discussed the data.

New data from a report on US Medicaid program spending suggests that spending on disease-modifying therapies has more than doubled over a recent 7-year stretch, with no expected help from generic competition coming soon.

Biohaven’s small molecule CGRP receptor antagonist rimegepant has displayed safety and efficacy in a small cohort of patients with migraine using it alongside erenumab.

In a review of 5 clinical trials, non‐vitamin‐K oral anticoagulants were shown to be associated with a lower risk of fatal and disabling, as well as non-disabling, stroke compared to warfarin.

Those achieving ≥50% response attained migraine reductions nearly double that of the full cohort, with data possibly providing context for setting realistic patient expectations regarding the spectrum of response.

The assistant professor of psychology at East Carolina University spoke about the need for patient-specific, and sometimes creative, approaches to improving adherence, which itself involves more than just medication.

New 52-week data from an extension study has suggested that recently FDA-approved ubrogepant (Ubrelvy; Allergan) is safe and tolerable, with minimal treatment-related adverse events with 2 doses assessed.

MONEAD study data suggest that AED drug exposure in infants is low compared with their mothers who were taking AED therapy, and thus supports the general safety of breastfeeding by mothers with epilepsy.

The guidelines included a review of 23 studies, suggesting that patients with normal neurological examinations do not require neuroimaging assessments as part of migraine treatment.

Recent findings suggest that patients with Parkinson disease may have impaired swimming ability, even in light of motor improvements with treatment.

In this Peer Exchange, experts discussed current SMA therapies, what they have learned, and what the future holds for treatment of this devastating disease.

The oral, dual-mechanism therapy from Axsome Therapeutics met both of its primary end points in the phase 3 MOMENTUM trial in acute migraine, and is expected to be filed with the FDA.

Catch up on some of our most popular Peer Exchange panels with experts in the field, including discussions on epilepsy, migraine, multiple sclerosis, and more.

After promising results in prior phase 2 study, Stealth BioTherapeutics’ elampretide has failed to show change in the 6-minute walk test and Primary Mitochondrial Myopathy Symptom Assessment total fatigue score in the MMPOWER-3 trial.

The behavioral director at the Angelman Syndrome Clinic at Massachusetts General Hospital offered insight into the challenges in studying a drug, such as gaboxadol, in Angelman syndrome.

The second of 2 identical trials investigating 2 doses of low-dose fenfluramine (Fintepla) is set to be completed in early 2020, as the agent awaits FDA approval.

Branded as Ubrelvy, Allergan’s oral calcitonin gene-related peptide antagonist is the first-in-class to be approved for this indication.

Panelists review the delicate process of introducing a new medication to a treatment regimen and the creative endeavors employed to improve adherence.

The behavioral director at the Angelman Syndrome Clinic at Massachusetts General Hospital discussed the phase 3 NEPTUNE trial of gaboxadol, also known as OV101.

After resubmitting the NDA for apomorphine sublingual film (APL-130277), the FDA has set the new PDUFA action date of May 21, 2020.

Compared to counterparts who did not exercise, those with cognitive impairments with no dementia risk factors improved in executive functioning when they exercised consistently over 6 months.

The third generation, high-affinity, selective small molecule CGRP receptor antagonist was significantly superior in the 2 higher doses on both co-primary end points of pain freedom and most bothersome symptoms.

Given the data, Adamas has stated that it will forgo its originally planned second phase 3 placebo-controlled study, instead continuing its open-label extension study and engaging the FDA to discuss ADS-5102’s regulatory pathway.