Electrical analog model study of water in the Yabucoa Valley, Puerto Rico; Phase 1, collecting preliminary data and assembling available data

In this phase 1 trial, inhibition of granulocyte-macrophage colony-stimulating factor (GM-CSF) was associated with clinically meaningful responses in 5 of 15 patients with relapsed or refractory chronic myelomonocytic leukemia (CMML). Preliminary data suggest that this approach may be tractable in CMML bearing activating NRAS mutations.

Download Full-text

066 Avxs-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update

Journal of Neurology Neurosurgery & Psychiatry ◽

10.1136/jnnp-2019-anzan.58 ◽

2019 ◽

Vol 90 (e7) ◽

pp. A22.1-A22 ◽

Cited By ~ 1

Author(s):

John W Day ◽

Claudia A Chiriboga ◽

Thomas O Crawford ◽

Basil T Darras ◽

Richard S Finkel ◽

...

Keyword(s):

Preliminary Data ◽

Muscular Atrophy ◽

Phase 1 ◽

Neuromuscular Disorders ◽

Ventilatory Support ◽

Genetic Diagnosis ◽

Survival Motor Neuron ◽

Open Label ◽

Phase 3 ◽

Pivotal Phase

IntroductionSMA1 is a neurodegenerative disease caused by bi-allelic survival motor neuron 1 gene (SMN1) deletion/mutation. In the phase 1 study, SMN GRT onasemnogene abeparvovec (AVXS-101) improved outcomes of symptomatic SMA1 patients. We report preliminary data of STR1VE, a pivotal study (NCT03306277) evaluating efficacy and safety of a one-time intravenous AVXS-101 infusion.MethodsSTR1VE is a phase 3, multicenter, open-label, single-arm study in SMA1 patients aged <6 months (bi-allelic SMN1 loss, 2xSMN2). Primary outcomes: independent sitting for ≥30 seconds (18 months) and survival (14 months). Secondary outcomes: ability to thrive and ventilatory support (18 months). Exploratory outcomes: Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) and Bayley Scales of Infant and Toddler Development scores.ResultsEnrollment is complete with 22 patients dosed. Mean age at symptom onset, genetic diagnosis, and enrollment was 1.9 (0–4.0), 2.1 (0.5–4.0), and 3.7 (0.5–5.9) months. At baseline, no patient required ventilatory/nutritional support, and all exclusively fed by mouth. Mean baseline CHOP-INTEND score was 32.6 (17.0–52.0), which increased 6.9 (-4.0–16.0, n=20), 10.4 (2.0–18.0, n=12), and 11.6 (-3.0–23.0, n=9) points at 1, 2, and 3 months. Updates will be provided at the congress.ConclusionsPreliminary data from STR1VE show rapid motor function improvements in SMA1 patients, paralleling phase 1 findings.

Download Full-text

Erosion and nutrient loss reduction with an alternative planting method for coffee (Coffea arabica).

The Journal of Agriculture of the University of Puerto Rico ◽

10.46429/jaupr.v92i3-4.2633 ◽

1969 ◽

Vol 92 (3-4) ◽

pp. 153-169

Author(s):

David Sotomayor-Ramírez ◽

John Ramírez-Ávila ◽

Edwin Más ◽

Gustavo A. Martínez

Keyword(s):

Puerto Rico ◽

Coffea Arabica ◽

Phase 1 ◽

Nutrient Loss ◽

Nutrient Concentrations ◽

Phase 2 ◽

Nutrient Losses ◽

Nutrient Runoff ◽

The Media ◽

Planting Method

Coffee (Coffea arabica) planting in the interior mountainous region of Puerto Rico is usually performed on steep slopes after vegetation removal. The construction of individual terraces around the tree, such as the "Media Luna" planting method, prior to planting could reduce sediment and nutrient losses and could increase yields by improved on-site water and nutrient retention. Experiments were conducted to test the hypothesis that the "Media Luna" planting technique could reduce sediment, total phosphorus (TP), and total Kjeldahl nitrogen (TKN) in runoff during and after the establishment of a coffee plantation in Puerto Rico. The experiments were conducted on a commercial farm where the predominating soils were Mucara (Dystric Eutrudepts) in Phase 1, and Humatas (Typic Haplohumults) in Phase 2. In Phase 1 (recent plantings), sediment and nutrient runoff losses were similar in the conventional countour planting method and in the "Media Luna" treatments. Nutrient concentrations in runoff increased in events following fertilization. Recently loosened unconsolidated sediment material in the "Media Luna" treatment may be more susceptible to losses during the initial establishment phase. In phase 2 (mature plantings), sediment and nutrient losses were greater from soils planted with the conventional contour planting method than from those with the "Media Luna" treatment. The lower TP concentrations measured during Phase 2 suggests that the "Media Luna" technique could be a beneficial practice for coffee production in some areas of Puerto Rico.

Download Full-text