For Patients

If you have scleroderma, you are not alone. The scleroderma community is made up of tens of thousands of patients and their loved ones worldwide. The SRF is here to help.

The first time many people hear about scleroderma is when they, a family member or friend are diagnosed with the disease. Scleroderma is a complex and surprisingly widespread illness, affecting as many people as more commonly recognized diseases such as multiple sclerosis and muscular dystrophy.

In addition to funding the most promising research aimed at improved therapies and a cure, the Scleroderma Research Foundation provides information that may help scleroderma patients better understand their disorder and more effectively manage its symptoms.

To learn more about the various forms and subtypes of scleroderma, please click here. This section of the Scleroderma Research Foundation’s Web site provides information for patients to educate themselves, as well as their caretakers and loved ones, about this serious disease.

Please remember, information provided on this Website and others is intended as a guide. Specific medical advice can only be provided by your health care professional.

 
 

Research News

EULAR: Updated scleroderma guidance focuses on new treatments, approaches

Author: Sara Freeman
Date Published: June-2015
Source: PM360

ROME (FRONTLINE MEDICAL NEWS) – Updated expert recommendations from the European League Against Rheumatism for the treatment of systemic sclerosis will focus on several new treatment options, although the use of biologic agents is not included in detail because there are still too few data on these drugs to give firm guidance on their use. The EULAR Scleroderma Trials and Research group (EUSTAR) recommendations for the treatment of scleroderma were first published 6 years ago ( Ann. Rheum. Dis. 2009;68:620-8 ) and considered data through December 2006. “Since then, a number of new drugs have become available and new and important information has been published concerning treatments already known before,” said Dr. Otylia Kowal-Bielecka of the Medical University of Bialystok, Poland, who discussed some of the main highlights of the revised recommendations (Ann. Rheum. Dis. 2015;74:90-1) at the European Congress of Rheumatology.

Fresolimumab treatment decreases biomarkers and improves clinical symptoms in systemic sclerosis patients

Author: L. Rice, Cristina M. Padilla, S. McLaughlin, A. Mathes, J. Ziemek, S. Goummih, S. Nakerakanti, M. York, G. Farina, M. Whitfield, R. Spiera, R. Christmann, J. Gordon, J. Weinberg, R. Simms, and R. Lafyatis
Date Published: June-2015
Source: Journal of Clinical Investigation

BACKGROUND. TGF-β has potent profibrotic activity in vitro and has long been implicated in systemic sclerosis (SSc), as expression of TGF-β–regulated genes is increased in the skin and lungs of patients with SSc. Therefore, inhibition of TGF-β may benefit these patients. METHODS. Patients with early, diffuse cutaneous SSc were enrolled in an open-label trial of fresolimumab, a high-affinity neutralizing antibody that targets all 3 TGF-β isoforms. Seven patients received two 1 mg/kg doses of fresolimumab, and eight patients received one 5 mg/kg dose of fresolimumab. Serial mid-forearm skin biopsies, performed before and after treatment, were analyzed for expression of the TGF-β–regulated biomarker genes thrombospondin-1 (THBS1) and cartilage oligomeric protein (COMP) and stained for myofibroblasts. Clinical skin disease was assessed using the modified Rodnan skin score (MRSS).

FDA Grants Breakthrough Therapy Designation for Actemra (tocilizumab) in Systemic Sclerosis

Author: Genentech
Date Published: June-2015
Source: Genentech

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to Actemra® (tocilizumab) for systemic sclerosis, also known as scleroderma.1 This designation is intended to expedite the development and review of medicines with early signals of potential clinical benefit in serious diseases and to help ensure patients have access to them as soon as possible. Genentech has also initiated a Phase III study in systemic sclerosis (NCT02453256), a disease for which there are inadequate treatment options.3,4

Cardiac troponin testing in idiopathic inflammatory myopathies and systemic sclerosis-spectrum disorders: biomarkers to distinguish between primary cardiac involvement and low-grade skeletal muscle disease activity

Author: Michael Hughes, James B Lilleker, Ariane L Herrick, Hector Chinoy
Date Published: March-2015
Source: Annals of the Rheumatic Diseases

Primary cardiac involvement, an under-recognised manifestation of the idiopathic inflammatory myopathies (IIM) and systemic sclerosis (SSc)-spectrum disorders, is associated with significant mortality. Within these two conditions, traditional skeletal muscle enzyme testing may not effectively distinguish between skeletal and cardiac muscle involvement, especially in patients with subclinical cardiac disease.

Adipocyte–myofibroblast transition: linking intradermal fat loss to skin fibrosis in SSc

Author: Sarah Onuora
Date Published: January-2015
Source: Nature Reviews Rheumatology

Myofibroblasts are considered the primary fibrogenic effector cells in systemic sclerosis (SSc), but the origin of these cells within fibrotic lesions is a matter of debate. A new study published in Arthritis & Rheumatology shows that the majority of dermal myofibroblasts in fibrotic skin arise from adiponectin-positive progenitors resident in the…

News for Patients

Women’s Immune System Genes Operate Differently From Men’s

Author: Jennie Dusheck
Date Published: July-2015
Source: Stanford Medicine

A new technology reveals that immune system genes switch on and off differently in women and men, and the source of that variation is not primarily in the DNA.A new technology for studying the human body’s vast system for toggling genes on and off reveals that genes associated with the immune system toggle more frequently, and those same genes operate differently in women and men. Some genes are virtually always on, like the clock light on a microwave; others sit unused for years at a time, like some regrettable appliance you bought, stuffed into the back of the closet and forgot. Some genes can be always on in one person and always off in another. A minority of genes switch on and off, like a favorite cell phone app. A new technology, which makes it possible to study the molecules that regulate all of that switching in living people as they go about their lives, has revealed some intriguing surprises, according to a study from the Stanford University School of Medicine.

Dangerous Clot Risks Higher in SSc – Especially in First Year after Diagnosis

Author: Rita Baron-Faust MPH
Date Published: July-2015
Source: Rheumatology Network

Patients diagnosed with systemic sclerosis (SSc) may have a three times greater risk of venous thromboembolism (VTE) – including potentially deadly clots in the lungs – and an increased risk in the first year after a diagnosis, according to a large population database study from Canada. The study of 1,245 SSc patients (83% female, mean age 56 years old),and 12,670 matched controls in British Columbia (BC), attributes the increased risk to vascular damage from SSc-related fibrosis that causes endothelial dysfunction and hypercoagulability due to inflammation.

Unique Microbial Signature Identified in Systemic Sclerosis

Author: Pam Harrison
Date Published: June-2015
Source: Medscape

Patients with systemic sclerosis have a distinct colonic microbiome that might contribute to clinical manifestations of the disease, according to a new study. "I see scleroderma patients exclusively in my practice, and gastrointestinal symptoms are sometimes the number one complaint," said Elizabeth Volkmann, MD, from the University of California at Los Angeles. "Patients often don't even feel comfortable disclosing these symptoms because they are so personal."

Tocilizumab and Systemic Sclerosis

Author: James Radke, PhD
Date Published: June-2015
Source: Rare Disease Report

t’s a busy week for Genentech and systemic sclerosis researchers. First, the US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation status to tocilizumab (Actemra) for treating patients with systemic sclerosis, or scleroderma.1 The designation should expedite the development and review of tocilizumab for this rare condition. Second, data from the phase 2 study that led to the Breakthrough Designation is being presented at EULAR 2015 this week as well. The developers of tocilizumab, Genentech also announced they have begun a phase 3 study to test the safety and efficacy of tocilizumab in patients with systemic sclerosis.

EULAR: Updated scleroderma guidance focuses on new treatments, approaches

Author: Sara Freeman
Date Published: June-2015
Source: PM360

ROME (FRONTLINE MEDICAL NEWS) – Updated expert recommendations from the European League Against Rheumatism for the treatment of systemic sclerosis will focus on several new treatment options, although the use of biologic agents is not included in detail because there are still too few data on these drugs to give firm guidance on their use. The EULAR Scleroderma Trials and Research group (EUSTAR) recommendations for the treatment of scleroderma were first published 6 years ago ( Ann. Rheum. Dis. 2009;68:620-8 ) and considered data through December 2006. “Since then, a number of new drugs have become available and new and important information has been published concerning treatments already known before,” said Dr. Otylia Kowal-Bielecka of the Medical University of Bialystok, Poland, who discussed some of the main highlights of the revised recommendations (Ann. Rheum. Dis. 2015;74:90-1) at the European Congress of Rheumatology.

Ways to Give

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