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September 2, 2019August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 29, 2019August 28, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

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Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 26, 2019August 26, 2019

Adalimumab May Safely Treat Sarcoidosis in Hepatitis C, Case Report Suggests

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People with hepatitis C who develop sarcoidosis may be safely treated with adalimumab, marketed by Abbvie as Humira, a case report suggests. The case was described in a study, “Sarcoidosis with ... Read more

August 20, 2019August 20, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

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Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

August 19, 2019August 19, 2019

Sarcoidosis Educational Event Set for Saturday at University of Cincinnati

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An educational conference for people with sarcoidosis and those close to them will be held at the University of Cincinnati this Saturday, featuring doctors with expertise in this disease, presentations, and ... Read more

August 12, 2019August 12, 2019

Analysis Highlights Importance of Risk Assessment in Cardiac Sarcoidosis

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A new statistical analysis of Finland’s nationwide cardiac sarcoidosis (CS) registry found that sudden cardiac death plays a significant role in the outcome of CS patients, affirming the importance ... Read more

August 5, 2019August 5, 2019

Jakafi Shows Promise for Treatment of Cutaneous Sarcoidosis, Report Says

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Using Jakafi (ruxolitinib) to treat a woman with polycythemia vera (PV; a blood cancer) led to complete resolution of her cutaneous sarcoidosis, and to mild reduction of her lung nodules, ... Read more

August 2, 2019August 2, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

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Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage ... Read more

August 1, 2019

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

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Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state ... Read more

July 29, 2019July 29, 2019

Rare Case of Spinal Sarcoidosis Provides Guidelines for Diagnosis, Management

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A new report describes a woman with a very rare case of sarcoidosis involving only the spine who entered full remission after surgical removal of the lesion followed by ... Read more

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