rare disease Latest News

Understanding Drug Development will be the second webcast in the RARE Webinar Series providing relevant information to patients and caregivers battling rare diseases, which are defined as diseases that affect fewer than 200,000 people in the US and...

CADASIL is one of approximately 7,000 rare diseases affecting 30 million Americans. CADASIL is now listed at the National Multiple Sclerosis Society as a condition a doctor needs to rule out before diagnosing a patient with MS.

The Fowler family recently relocated to the Chicago area to get specialized medical treatment for their son with Hunter Syndrome. Filmmaker Joey Howell will film them as a part of his documentary at Lurie Children's Hospital on October 16/17.

To learn how to raise more awareness of and funding for research about her son's rare disease, Hunter Syndrome, mom Melissa Hogan wrote a moving essay to win a scholarship to the Mayo Clinic's Social Media Week events.

Customer and company experts to discuss identifying causative mutations using arrays and sequencing

Atlanta Motivational Speaker Mandy Young Will Inspire Crowds Nationwide As The Latest Addition To The Motivational Day Group Of Speakers

NBIA Disorders Association will join the National Organization for Rare Disorders in observing Rare Disease Day on Wed., Feb. 29 to focus attention on patients affected by rare diseases, like Neurodegeneration with Brain Iron Accumulation (NBIA)

The Board of Directors of the Caring for Carcinoid Foundation is pleased to announce the appointment of Ron Hollander as its new Executive Director.

Understanding Drug Development will be the second webcast in the RARE Webinar Series providing relevant information to patients and caregivers battling rare diseases, which are defined as diseases that affect fewer than 200,000 people in the US and...

Jasper Against Batten at Cures Within Reach, a 501(c)3 not-for-profit located in IL, is collaborating with an int'l coalition to fund “Global Gene Transfer for Batten Disease” research at the UNC Gene Therapy Center, announced Dr. Bruce Bloom.

Each year the designation of the final day of February as Rare Disease Day generates increased awareness of the 7,000-plus rare disorders and fuels a greater urgency to develop more treatments for rare disease patients.

CADASIL is one of approximately 7,000 rare diseases affecting 30 million Americans. CADASIL is now listed at the National Multiple Sclerosis Society as a condition a doctor needs to rule out before diagnosing a patient with MS.

After years of development and repeated rejections, uniQure finally got permission to market its gene therapy—Glybera.

The Fowler family recently relocated to the Chicago area to get specialized medical treatment for their son with Hunter Syndrome. Filmmaker Joey Howell will film them as a part of his documentary at Lurie Children's Hospital on October 16/17.

For the past year, documentary filmmaker Joey Howell has been filming a documentary called Boys with Bigger Hearts about the rare disease Hunter Syndrome. The project is raising funds via Kickstarter to finish the filming and production.

To learn how to raise more awareness of and funding for research about her son's rare disease, Hunter Syndrome, mom Melissa Hogan wrote a moving essay to win a scholarship to the Mayo Clinic's Social Media Week events.

Customer and company experts to discuss identifying causative mutations using arrays and sequencing

Atlanta Motivational Speaker Mandy Young Will Inspire Crowds Nationwide As The Latest Addition To The Motivational Day Group Of Speakers

The Anderson Cooper Daytime Talk Show will feature a segment titled " The Boy Who Cannot Eat", discussing Eosinophilic Disorders on Friday, March 23, 2012

APFED, representing those living with eosinophil associated disorders, joins NORD to create awareness of rare diseases on Rare Disease Day, February 29, 2012.

NBIA Disorders Association will join the National Organization for Rare Disorders in observing Rare Disease Day on Wed., Feb. 29 to focus attention on patients affected by rare diseases, like Neurodegeneration with Brain Iron Accumulation (NBIA)

The Board of Directors of the Caring for Carcinoid Foundation is pleased to announce the appointment of Ron Hollander as its new Executive Director.

DrugPatentWatch.com announces new Orphan Drug Exclusivity report

Jacob's Cure participating in Chase Community Giving Project. Choose Jacob's cure as the charity you support and help ensure Jacob;s spot as a top 200 charity

W. Mark Dendy has too much, and he is trying to get rid of it! Dendy, author of "The Cascade Effect: A Biologist Shares His Faith and His Story Living with Wilson's Disease" has a rare genetic condition that results in toxic copper buildups.

A weekend of special events, featuring a 4-time Olympic athlete, is coming to the North Mecklenburg / Concord area on October 3rd and 4th – you don’t want to miss this!

An estimated 3 to 8 million people have symptoms of “interstitial cystitis” (IC or Painful Bladder). IC causes frequent urination, pelvic pain, and urinary urgency. Treatments are evolving including proof that food plays a key role in symptoms.

The Penn State Football Uplifting Athletes fifth annual Lift For Life® event was a tremendous success.