Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study

Background The development and approval of disease modifying treatments have dramatically changed disease progression in patients with spinal muscular atrophy (SMA). Nusinersen was approved in Europe in 2017 for the treatment of SMA patients irrespective of age and disease severity. Most data on therapeutic efficacy are available for the infantile-onset SMA. For patients with SMA type 2 and type 3, there is still a lack of sufficient evidence and long-term experience for nusinersen treatment. Here, we report data from the SMArtCARE registry of non-ambulant children with SMA type 2 and typen 3 under nusinersen treatment with a follow-up period of up to 38 months. Methods SMArtCARE is a disease-specific registry with data on patients with SMA irrespective of age, treatment regime or disease severity. Data are collected during routine patient visits as real-world outcome data. This analysis included all non-ambulant patients with SMA type 2 or 3 below 18 years of age before initiation of treatment. Primary outcomes were changes in motor function evaluated with the Hammersmith Functional Motor Scale Expanded (HFMSE) and the Revised Upper Limb Module (RULM). Results Data from 256 non-ambulant, pediatric patients with SMA were included in the data analysis. Improvements in motor function were more prominent in upper limb: 32.4% of patients experienced clinically meaningful improvements in RULM and 24.6% in HFMSE. 8.6% of patients gained a new motor milestone, whereas no motor milestones were lost. Only 4.3% of patients showed a clinically meaningful worsening in HFMSE and 1.2% in RULM score. Conclusion Our results demonstrate clinically meaningful improvements or stabilization of disease progression in non-ambulant, pediatric patients with SMA under nusinersen treatment. Changes were most evident in upper limb function and were observed continuously over the follow-up period. Our data confirm clinical trial data, while providing longer follow-up, an increased number of treated patients, and a wider range of age and disease severity

Widening the Application Range of PLA‐Based Thermoplastic Materials through the Synthesis of PLA‐Polyether Block Copolymers: Thermal, Tensile, and Rheological Properties

Abstract Polyethylene glycol (PEG), polypropylene glycol (PPG), and poly(ethylene glycol‐co‐propylene glycol) (PEPG) of number average molar masses (Mn) of 6–20 kDa are used as macroinitiators in the ring‐opening polymerization (ROP) of l‐lactide to synthesize high molar mass (50 kDa < MnCopo < 120 kDa) PLLA‐b‐polyether‐b‐PLLA linear triblock and starblock copolymers. At the studied block lengths, PEG and PEPG blocks exhibit miscibility with the PLLA blocks in the amorphous domains leading to a plasticization effect. As the PEG or PEPG block content is increased to 18 %wt, the Tg (Tg ≈ 24 °C) and the elastic modulus (E ≈ 500 MPa) are reduced, while the elongation at break (εb ≈ 280%) and crystallization rate are increased. At the same time, small angle oscillatory shear (SAOS) rheometric measurements show that the plasticized copolymers have a reduced melt viscosity. In contrast, SAOS and DSC measurements of the PPG‐containing block copolymers reveal phase separation of the PPG and PLA blocks leading to microstructures in the melt. Tensile tests show that the phase‐separated PPG‐containing block copolymers are more ductile than PLA homopolymers, but more brittle than the PEG‐ or PEPG‐plasticized block copolymers

Melt spinnable copolymers from polyacrylonitrile, method for producing fibers or fiber precursors by means of melt spinning, and fibers produced accordingly

Die Erfindung betrifft ein Verfahren zur Herstellung von thermisch stabilisierten schmelzgesponnenen Fasern, bei dem durch Schmelzspinnen hergestellte Polyacrylnitril (PAN)‐Fasern bzw. PAN‐Faserprecursoren in einer wässrigen alkalischen, zusätzlich ein Lösungsmittel für PAN enthaltenden Lösung behandelt werden. Ebenso betrifft die Erfindung Fasern, die gemäß diesem Verfahren herstellbar sind

Online monitoring of polymer nanoparticle growth as valuable tool in the development of adhesive dispersions: Presentation held at In-Adhesives Symposium, Munich, 15th of February 2017

The efficient implementation of synthesis concepts into robust and reproducible emulsion polymerization processes requires a reliable process control, ideally also with respect to an inline monitoring of polymer nanoparticle growth. If critical process parameters like e.g. emulsification of the monomers, particle formation, colloidal stability during processing, heat flux, and viscosity are monitored inline and continuously, an increased understanding of the polymerization process is achieved, resulting in safe and cost-optimal processes and feedback control strategies. For example, polyvinylacetate based dispersion adhesives (white glues) are usually modified with functionalized comonomers to control glue properties like viscosity, pH, film formation, hydrophobicity, and adhesive strength (via crosslinking). However, the modification will also influence process characteristics like particle nucleation and growth or polymerization rates. Technologies which provide information about these features might improve process control and hence product quality. To date only a limited number of process analytical technologies, suitable for high concentrations of nanoparticles, exist. The recently developed Photon Density Wave (PDW) spectroscopy allows for the precise and calibration-free characterization of the optical properties of particles and droplets during their processing. Its fundamental benefit - the quantitative separation of light absorption and light scattering - enables particle sizing also in highly concentrated polymer dispersions (> 40 vol%), in diameter ranges of approx. 50 nm 500 µm. The contribution will introduce PDW spectroscopy and discuss its benefits based on the monitoring of polymerization processes like the synthesis of highly concentrated functionalized polyvinylacetate adhesives

Verfahren zur Herstellung von thermisch stabilen schmelzspinnbaren PAN-Copolymeren, PAN-Copolymere, hieraus gebildete Formkörper sowie Verfahren zur Herstellung dieser Formkörper

Die vorliegende Erfindung betrifft einen neuen Synthese-Prozess, um schmelzbare PAN-Copolymere mit erhöhter thermischer Stabilität herzustellen. Die gemäß dem erfindungsgemäßen Verfahren hergestellten PAN-Copolymere zeigen überraschenderweise verbesserte thermische und mechanische Eigenschaften, verglichen mit gemäß aus dem Stand der Technik bekannten Methoden hergestellten PAN-Copolymeren. Zudem betrifft die vorliegende Erfindung entsprechende PAN-Copolymere sowie hieraus hergestellte Formkörper bzw. ein Herstellungsverfahren zur Herstellung derartiger Formkörper, insbesondere schmelzgesponnener Mono- oder Multifilamente

Novel polyelectrolytes with regular structure-synthesis, properties and applications

A review, with 17 refs., on prepn. and properties and uses of polyelectrolytes based on poly(4-vinylpyridine) via functionalization. Cationic polyelectrolytes and polymeric betaines with narrow mol. wt. distribution and styrene block copolymers contg. charged and uncharged blocks of different hydrophilicity/hydrophobicity are described. The block copolymers are useful stabilizers in pptn. and emulsion polymn. processes. [on SciFinder (R)