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In This Issue
Drug Pipelines Improving
FDA Safety and Innovation Act
Juvenile-Onset Systemic Sclerosis
SRF Webinar Series
Your Voice Matters!
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Insights eNewsletter

Dear ,


Thanks to everyone in the scleroderma community who participated in raising awareness during Scleroderma Awareness Month. June 2012 saw blue awareness bracelets worn by many, new stories on the SRF Hero Wall as well as many special events hosted by families and individuals across America.

More effort is needed to spread the word about scleroderma and the vital role medical research plays in the search for new therapies and a cure. Collectively our voice is strong and we encourage you to help carry the momentum forward. Lend your voice by asking elected officials to support scleroderma-specific legislation, sharing the Insights e-Newsletter or asking friends to join us on Facebook. Every action takes us closer to a cure! 
Drug Pipelines Improving After Years in Doldrums

Data from Thomson Reuters published on Tuesday showed more new drugs are reaching the market and that the industry has been enjoying higher success rates in the costly final stage of clinical development.

The number of innovative medicines, or new molecular entities, launched globally in 2011 hit a 10-year high of 31, up from 21 in 2010.


Last year's entrants included significant advances, such as new treatments from Roche and Bristol-Myers Squibb for melanoma, the first new drug for lupus in half a century from GlaxoSmithKline, and two new hepatitis C drugs from Merck and Vertex Pharmaceuticals.

The outlook is also improving for the next wave of products, with the number of experimental drugs submitted last year for regulatory approval - the final hurdle in getting to market - nearly double the 2007 level.   


Furthermore, the tally of drugs terminated in the final Phase III stage of clinical trials dropped markedly, to 45 in the last three years from 53 in 2008-10, according to the 2012 Pharmaceutical R&D Factbook from Thomson Reuters unit CMR International.  Read more...  


Visit our site to learn more about the currently available treatments for scleroderma patients.  

FDA Safety and Innovation Act: A Step Forward for Patients With Rare Diseases
The Food and Drug Administration Safety and Innovation Act (FDASIA) includes the most groundbreaking measures for rare disease patients and their families since the Orphan Drug Act of 1983.  And, just as the rare disease patient community-with NORD as its unifying voice-played a major role in the Orphan Drug Act, NORD and rare disease patients have taken a leadership role in developing and shaping this new legislation and in securing its enactment in a timely manner.

Crafted for the purpose of reauthorizing the Prescription Drug User Fee Act (PDUFA), the FDA Safety and Innovation Act includes far-ranging provisions of critical importance to rare disease patients.  These reflect, in many cases, years of advocacy, education and relationship-building by NORD and its member organizations on behalf of patients. Read more... 

Juvenile-Onset Systemic Sclerosis Resembles Adult-Onset Disease
The pattern and course of disease in patients who develop systemic sclerosis in childhood or adolescence is similar to that in patients who develop the disease as adults, data from Europe has shown.

There were 60 patients among 5,000 included in the EUSTAR database (1.2%) who developed systemic sclerosis before the age of 16. Their mean age of onset was 12.4 years. They were compared with another 910 patients who first developed symptoms between the ages of 20 and 40, at a mean of 32 years.

Both groups had a mean disease duration of 17 years, and the illness was active at the last follow-up in about one-quarter of cases. The distribution of disease subsets was similar in juvenile-onset and adult-onset cases: roughly 40% had diffuse disease, 50% had limited disease and 10% had overlap syndrome. Read more...

Click here to read more news for patients.
SRF Webinar Series  

If you missed last month's webinar hosted by SRF Board Member and actor/director Bob Saget, it's available for viewing by clicking here. The live webinar, the fifth in an ongoing series, features an unscripted discussion with Bob and SRF Board Chair Luke Evnin. Topics ranged from how Bob became involved with the Foundation to the future of stem-cell treatment for scleroderma.


All previous webinars are also available on the SRF Website. Our next webinar will be held in August and details will be available soon. 

Speak Up! Your Voice Matters.

Here's a summary of June's Scleroderma Awareness Month Survey:


Sixty-six percent of respondents first learned of the disease when they (40%) or a friend or family member (26%) were diagnosed. The primary takeaway from this statistic is that there is much work to be done to raise the profile of this disease. Increased awareness of scleroderma will result in a better understanding of disease complications, heightened interest from policymakers and drug companies as well as more funding for vital medical research. Fourteen percent of people first learned of scleroderma through Bob Saget's made-for-television movie, For Hope, about his sister's struggle with the disease, starring Dana Delany and featuring SRF Founder Sharon Monsky.


Almost half of all those responding (49%) know only one person with scleroderma, whether it be themselves or a friend/family member. This is to be expected since scleroderma is a rare disease. However, in a clear showing of how connected this community is becoming, nearly 26% now know more than 10 people with the disease.


As another example of how active the community is becoming, more than 32% of respondents met others with scleroderma online. Twenty-six percent met others at support group meetings. Nearly 12% met other patients through introductions by friends or family members and 6% have happened upon another patient in a public setting. The remaining respondents either haven't met anyone with scleroderma or they have met others at a fundraising or awareness-building activity like a Cure Crew event.


Answers to the question "What do you wish people knew about scleroderma?" were largely consistent with the following statement: "I wish more people knew about scleroderma; how it's much more than a skin disease, can be deadly and has changed my life in so many complicated ways." Demonstrating scleroderma's myriad symptoms, some people wish that others would not judge them by their affected appearance while others wished the opposite: that people understood despite limited change in physical attributes, the internal changes are extraordinary and debilitating.


Survey participants feel that the number one way to increase scleroderma awareness is through social media like Facebook, Twitter, Instagram and others. The next best methods, in order of anticipated success rate, are (2) educational events; (3) word of mouth; (4) advertising and fundraising events (tied); and (5) public service announcements on television or radio.


Most people asked that the SRF continue and to step up efforts to raise awareness by asking celebrities to be more involved, considering new advertising and public relations opportunities, finding new ways to put scleroderma "in the news" and continuing to distribute new information via every possible distribution channel. We're listening!


Lastly, we thank respondents for recognizing that they are a vital part of the solution. Increasing social media postings about scleroderma, bringing new people to the SRF website or Facebook page as well as hosting awareness and fundraising events in local communities are all excellent ways to expedite progress toward new therapies and a cure.


This month's poll: Coping with Scleroderma. What are the unique ways in which you cope with scleroderma symptoms? Share your response here. We'll share selected responses in next month's e-newsletter.  

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