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In This Issue
World Scleroderma Day
SRF Webinar Series
A Healthy Tension in Translational Research
Positive Results For New Oral PAH Drug
Systemic Sclerosis Stem Cell Transplant
NCATS Researchers Propose Innovative Approach
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World Scleroderma Day - Sunday June 29, 2014  


June is Scleroderma Awareness Month and Sunday, June 29 is World Scleroderma Day. The Scleroderma Research Foundation has partnered with the Scleroderma Foundation and the Scleroderma Society of Canada to help educate and raise awareness. Join us and other organizations around the globe to put a spotlight on scleroderma. What will you do to help find a cure? Here are a few suggestions:   


  • Share the Project Scleroderma video with family, friends, colleagues and media outlets.  
  • Honor your scleroderma heroes with a post on Facebook, Twitter, Instagram or  
  • Make sure to join the social media campaign this Sunday on Twitter and Facebook to make your voice heard. Everything helps!


World Scleroderma Day focuses on the need for heightened public awareness promoting a greater understanding of the disease and the need for increased funds for research into the underlying causes of and ultimately a cure for scleroderma.

SRF Webinar Series 
Next up in the SRF webinar series Dr. Francesco Boin will present live on Thursday, August 21 at 1:00 PM EDT from The Johns Hopkins Scleroderma Center of Excellence. Dr. Boin will provide his expert insights on Diagnosis and Early Management of Scleroderma. His session will take viewers from diagnosis through management of complications that impact the newly diagnosed as well as those who have been living with the disease for many years.

You can register here: https://cc.readytalk.com/r/q1qvpw5wsdxc&eom 


Dr. Boin is a physician-scientist; his research focuses on the cellular biology of immune cells and the mechanisms of autoimmunity in scleroderma. His goal is to identify reliable laboratory tools that can help investigate the causes of scleroderma, effectively measure disease activity, monitor treatments and help predict clinical outcomes in scleroderma patients.


The SRF webinar series provides the scleroderma community with access to leading experts in the field and information that is relevant to better managing the disease. As stated by a recent attendee, "[The webinar] empowers me to have additional discussion with my rheumatologist." 

A Healthy Tension in Translational Research 
A somewhat simplistic but nevertheless informative categorization of science collapses the principles employed to two basic approaches: inductive and deductive reasoning. While both seek to derive truth, the former involves an iterative process of hypothesis and refinement based on experimental observation, while the latter applies rigorous logic to derive the inescapable consequences of fundamental principles that are deemed absolute. Given the daunting complexity of biological systems, it remains true that investigators in the biomedical sciences are largely restricted by necessity to the realm of inductive scientific reasoning. Absolute truths, while conceivable, remain elusive and inherently constrained by the nature of the system used to achieve them.
Positive Results For New Oral Drug From Actelion 

Actelion has announced positive top-line results for a phase III trial of a new oral drug for the treatment of pulmonary arterial hypertension (PAH). The drug, selexipag, is a first-in-class orally available selective prostacyclin IP receptor agonist.

The pivotal, double-blind GRIPHON study was the largest outcome trial ever performed in PAH. 1,156 PAH patients were randomized to selexipag or placebo. The company said that trial had "met its primary efficacy endpoint with high statistical significance." Read more...

Systemic Sclerosis Stem Cell Transplant Risky but Promising

Autologous hematopoietic stem cell transplantation (HSCT) can produce durable event-free survival for patients with diffuse cutaneous systemic sclerosis (SSc) who are able to survive the procedure, but the type of pretransplant cardiac workup commonly used with HSCT in oncology is not sufficient to identify those who are unlikely to survive HSCT long enough to benefit, according to a new study published online June 24 in JAMA.

Coauthor Alois Gratwohl, MD, told Medscape Medical News, "There is now a treatment modality available for patients with severe systemic sclerosis, which can alter the devastating natural course of the disease. HSCT should be envisaged very early on; HSCT cannot change already-existing irreversible organ damage." Dr. Gratwohl is professor emeritus, former head of hematology, and former head of the stem cell transplantation team, Basel University Hospital, Switzerland.Know your target audience. Read more (free log-in required)...


NCATS Researchers Propose Innovative Approach to Test Drugs in Rare Diseases

Developing effective treatments is a slow and costly process, and more than 80 percent of investigational drugs tested in clinical trials fail. Discovering treatments for rare diseases can be even more challenging because the small numbers of patients with these diseases make it difficult to find enough people who can participate in clinical trials. The small numbers of rare disease patients also make gathering information about the diseases difficult. As a result, scientists often know little about the symptoms and biology of these conditions, which adds to the complexity of designing drug studies.  
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