References in periodicals archive ?
A number of factors, including (i) whether Catalyst's proof-of-concept study evaluating Firdapse for the treatment of SMA Type 3 will be successful, and (ii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2018 and its other filings with the U.S.
Additional presentations at the two meetings highlight results from the ongoing open-label SHINE extension study of children with infantile and later-onset SMA as well the CS2/CS12 analysis of older patients.
There are three management options for the SMA dissection, with conservative being the most commonly and initially used approach, provided the patient is stable.
While training and supporting its team of full-time staff is an essential part of SMA's mission, 2018 also saw SMA welcome a total of 265 volunteers and 69 interns from the local community, schools, universities and other entities who discovered the extensive and varied work carried out by SMA across its museums and various community activations.
Among these, nusinersen is the first drug specifically approved to treat SMA. Prior to approval in Europe, nusinersen was provided to patients with SMA type 1 within an Expanded Access Program (EAP).
On the march Last year's Sma' Shot parade in Paisley town centre
Chorion villus sampling (CVS) and amniocentesis (AC) are invasive prenatal tests (IPTs) used for the prenatal diagnosis of SMA. [10] Due to the recessive inheritance of types 1 to 4 SMA cases, up to 25% of prenatal diagnoses are expected to be SMA and pregnancies are terminated.
Figures 3 and 4 show the stress and strain curves of the superelastic SMA plate and the steel plate, respectively, obtained in accordance with the American Society for Testing and Materials (ASTM) guidelines so as to confirm the material property [27, 28].
Only diseases with accepted treatments can be added to the list, and parents like Resendez are hopeful that the FDA approval of Spinraza will bolster their argument for adding SMA. The committee will also discuss other conditions not on the Texas list, including Pompe disease, MPS-1 and X-ALD.
Spinraza is the first approved treatment in the European Union for SMA, a leading genetic cause of death in infants that is marked by progressive, debilitating muscle weakness.
SMA supplies more than 15,000 agricultural parts, primarily implement replacement parts and accessories, while Tisco offers approximately 16,000 tractor, combine, and agriculture equipment parts.