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.

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Research News

Evidence-based detection of pulmonary arterial hypertension in systemic sclerosis: the DETECT study

Author: J Gerry Coghlan,Christopher P Denton,Ekkehard Grünig,et al
Date Published: May-2013
Source: Annals of the Rheumatic Diseases

Objective: Earlier detection of pulmonary arterial hypertension (PAH), a leading cause of death in systemic sclerosis (SSc), facilitates earlier treatment. The objective of this study was to develop the first evidence-based detection algorithm for PAH in SSc.

Conclusions The novel, evidence-based DETECT algorithm for PAH detection in SSc is a sensitive, non-invasive tool which minimises missed diagnoses, identifies milder disease and addresses resource usage.

Systemic sclerosis (scleroderma, SSc) is a vascular disease

Author: Marco Matucci-Cerinic, Bashar Kahaleh, Fredrick M. Wigley
Date Published: May-2013
Source: Arthritis & Rheumatism

Molecular Signatures in Skin Associated with Clinical Improvement during Mycophenolate Treatment in Systemic Sclerosis

Author: Monique Hinchcliff, Chiang-Ching Huang, Tammara A Wood, J Matthew Mahoney, et al
Date Published: May-2013
Source: Journal of Investigative Dermatology

Heterogeneity in systemic sclerosis (SSc) confounds clinical trials. We previously identified “intrinsic” gene expression subsets by analysis of SSc skin. Here we test the hypotheses that skin gene expression signatures including intrinsic subset are associated with modified Rodnan skin score (MRSS) improvement during mycophenolate mofetil (MMF) treatment. Gene expression and intrinsic subset assignment were measured in 12 SSc patients’ biopsies and 10 controls at baseline, and from serial biopsies of 1 cyclophosphamide-treated patient and 9 MMF-treated patients.

The degree of skin involvement identifies distinct lung disease outcomes and survival in systemic sclerosis

Author: Tricia R Cottrell, Robert A Wise, Fredrick M Wigley & Francesco Boin
Date Published: April-2013
Source: Annals of the Rheumatic Diseases

Abstract - Objective To determine whether the pattern of skin involvement can predict clinical features, risk of restrictive lung disease (RLD) and survival in a large scleroderma (SSc) cohort. Methods Demographic and clinical data collected over 30 years from 2205 patients with SSc were retrospectively analysed after subdividing subjects into four subtypes based on pattern of skin fibrosis: type 0 (no skin involvement), type 1 (limited to metacarpophalangeal joints), type 2 (distal to elbows/knees) and type 3 (proximal to elbows/knees). Clinical features associated with skin subsets were identified by regression analyses. Kaplan–Meier and Cox proportional hazards models were used to compare time to RLD and survival across subtypes.

Open label study of escalating doses of oral treprostinil diethanolamine in patients with systemic sclerosis and digital ischemia: pharmacokinetics and correlation with digital perfusion.

Author: Shah AA, Schiopu E, Hummers LK, Wade M, Phillips K, Anderson C, Wise R, Boin F, Seibold JR, Wigley F, Rollins KD.
Date Published: April-2013
Source: Arthritis Research & Therapy

Abstract - INTRODUCTION: Treprostinil diethanolamine is an innovative salt form of the prostacyclin analogue, treprostinil sodium, developed as an oral sustained release (SR) osmotic tablet. The availability of a formulation permitting convenient systemic delivery might have applicability to scleroderma vascular complications. We evaluated pharmacokinetics and perfusion in scleroderma patients with digital ischemia following escalating twice-daily doses of treprostinil diethanolamine SR. METHODS: In this dual-center, open-label, phase I pharmacokinetic study, scleroderma patients with digital ulcers were enrolled. Drug concentrations and perfusion, quantified by laser Doppler imaging, were measured over 12 hours at the 2mg and 4mg (or maximally tolerated) doses.

News for Patients

Evidence-based detection of pulmonary arterial hypertension in systemic sclerosis: the DETECT study

Author: J Gerry Coghlan,Christopher P Denton,Ekkehard Grünig,et al
Date Published: May-2013
Source: Annals of the Rheumatic Diseases

Objective: Earlier detection of pulmonary arterial hypertension (PAH), a leading cause of death in systemic sclerosis (SSc), facilitates earlier treatment. The objective of this study was to develop the first evidence-based detection algorithm for PAH in SSc.

Conclusions The novel, evidence-based DETECT algorithm for PAH detection in SSc is a sensitive, non-invasive tool which minimises missed diagnoses, identifies milder disease and addresses resource usage.

Pulmonary hypertension often misdiagnosed, research finds - U. of C. study stresses need to identify condition earlier

Author: Jessica Tobacman
Date Published: May-2013
Source: The Baltimore Sun

Whitney Gaspar has long known that she didn't have much endurance. When she was in secondary school, Gaspar said she ran a "slow mile" in gym class of 14 to 15 minutes and "avoided stuff (that required) a lot of endurance." Then in December 1999, she caught the flu and couldn't walk four or five steps without her lips turning blue, said Gaspar, 36, of Plainfield. Doctors misdiagnosed Gaspar as having asthma. In February 2000, she finally got the correct diagnosis:

Actelion gets go-ahead to continue new drug trial

Author: Caroline Copley
Date Published: May-2013
Source: Reuters

Actelion should continue a late-stage study into a new heart and lung drug, independent monitors have recommended, with final results now expected by mid-2014, giving the biotech firm hope it has a further product in its pipeline. Actelion said in a statement the Independent Data Monitoring Committee had told the company it had unanimously recommended the continuation of a late-stage study in selexipag with no modifications, adding final results should come next year.

The FDA Wants You!

Author: Melissa Healy
Date Published: April-2013
Source: Los Angeles Times

The Food and Drug Administration, acting on a law signed by President Obama in 2012, has launched the third phase of an initiative to increase patient participation in the regulation of drugs and medical devices. On Wednesday, the agency went live with a new website aimed at demystifying the regulatory process for consumers and patient advocates. The new FDA website, called FDA Patient Network, is designed to educate consumers on the process of getting medical devices and drugs from the idea stage to pharmacy and hospital shelves. Along with the FDA Commissioner's blog, this is the agency's bid to be more transparent.

Faster Drug Approvals a Tonic for Pharma Industry

Author: Ben Hirschler
Date Published: May-2013
Source: Reuters

A pickup in new drug approvals, the promise of faster regulatory decisions and more targeted medicines have quickened the pulse of the pharmaceutical industry as a big wave of patent expiries recedes. Manufacturers are producing more targeted medicines, designed to treat very specific groups of patients, thanks to a new understanding of the genetic basis of many diseases - most notably cancer.

Ways to Give

There are many ways that you can support the work of the Scleroderma Research Foundation. We are grateful for your commitment to helping the SRF fund research that will result in improved therapies and, ultimately, a cure.

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