Author Archives: Ana de Barros PhD

BBrm02: BioBlast Pharma’s Novel Approach To Combat SMA

In a recent press release BioBlast Pharma Ltd., a clinical-stage orphan disease-focused biotechnology company, announced positive results of its preclinical proof-of-concept studies, conducted both in vitro and in vivo, for the company’s premier drug candidate BBrm02 to treat patients with Spinal Muscular Atrophy (SMA). BBrm02 belongs to a family of small molecule non-glycoside,…

Cure SMA Publishes New Booklet on Clinical Trials

An increasing amount of research on drugs to treat spinal muscular atrophy (SMA) is successfully progressing as a consequence of clinical trials, and Cure SMA believes there is a need to inform and educate the community about the clinical trial process. To do so, the nonprofit organization…

Isis Pharmaceuticals Announces Results From Phase 2 Study of ISIS-SMNRx In Infants With SMA Type I

Isis Pharmaceuticals, a Ribonucleic acid-targeted (RNA-targeted) drug discovery and development company, recently updated the results from an ongoing open-label Phase 2 trial of ISIS-SMNRx in infants with a diagnosis of spinal muscular atrophy (SMA) type I. The company had previously reported results from this trial which included assessments of muscle function,…

Cure SMA Releases Updates On SMA Drug Discovery Programs

Cure SMA, a non-profit organization focused on supporting research projects to find a cure for spinal muscular atrophy (SMA), has released an update on its drug pipeline of products currently under development to treat SMA. The organization has revealed its enthusiasm with both the increased number of projects…

Over $100,000 Raised at New England Walk-n-Roll to Help SMA

Over 600 walkers and runners helped raised more than $100,000 during the 15th Annual New England Chapter Walk-n-Roll, on Saturday, May 16, at the DCR Wompatuck Park in Hingham, MA. The participants in the event walked through 1.5 miles to support patients who suffer from spinal muscular atrophy (SMA), gathering money to fund research to…

NIH Funds Cure SMA’s Researcher Meeting

The non-profit organization Cure SMA has been awarded a National Institutes of Health (NIH) grant to support the preparation of its SMA Researcher Meeting. This is the seventh consecutive year that the NIH has financed the conference, and the funds will be used to support registration and travelling expenses of young fellow investigators who…

Discussion Draft on Compassionate Drug Use for Disease Such as SMA Released

A 21st Century Cures discussion draft has been released by the U.S. House of Representatives Energy and Commerce Committee as a basis for discussing new methods of accelerating discovery, development and delivery of novel therapies and cures. The initiative was established to investigate different ways the government can support these advancements. The newly revised and expanded…

Gene Therapy Research Awarded Additional $445,000 Cure SMA Grant

Nonprofit organization Cure SMA has awarded a $445,000 grant to fund research on a gene therapy for the treatment of spinal muscular atrophy (SMA) conducted at Nationwide Children’s Hospital. The additional funding was granted to the principal investigator of the Center for Gene Therapy, Brian Kaspar, PhD, as announced by the organization in a recent …

PTC Releases Data On New SMA Drug

PTC Therapeutics recently announced the release of clinical data from their Phase I trial testing the effects of the company’s oral formulation RG7800 to improve the status of spinal muscular atrophy (SMA) patients. The study entitled “SMN2 splicing modifier RG7800 shows dose-dependent increase of full length SMN2 mRNA in first-in-human study” was…

Cure SMA Releases Booklet on Musculoskeletal System

The nonprofit organization Cure SMA has released another one of its booklets focused on improving care for patients with spinal muscular atrophy (SMA). This edition, which is entitled “The Musculoskeletal System“ and included in the organization’s care series booklets, aims to increase knowledge on…