Global Genes Announces Fourth Annual RARE Tribute to Champions of Hope Award Recipients

July 15, 2015

Global Genes™ is pleased to announce the award recipients that will be recognized and honored at the 4th Annual RARE Tribute to Champions of Hope on September 26, 2015, at the Hyatt Regency Huntington Beach Resort and Spa in Huntington Beach, CA. The annual blue carpet event brings together scientists, advocates, patients and supporters who are leading the efforts in the fight against rare disease.

Over 300 individuals and organizations worldwide were nominated by their peers for a RARE Champion of Hope award for their notable efforts in rare disease advocacy, science, collaborative sciences, medical care and treatment. From the extraordinary list of nominees, members from the Global Genes Board of Directors, Medical and Science Advisory Board, and other key partners, selected the recipients to be honored at the 4th annual Tribute event.

Additionally, The 4th Annual RARE Patient Advocacy Summit™ will take place the two days prior to the Tribute, September 24 – 25. The Summit helps patient advocates become successful activists and offers a foundation to help patient advocates navigate their rare disease journey. You can expect many well recognized people and organizations participating on the 2015 summit agenda which include: Grace Whiting, National Alliance for Caregiving, Dr. Gayatri Rao, Director, Office of Orphan Products Development, FDA, David Glazer, Google Genomics, Brae Patrick-Lake Director, Stakeholder Engagement, Clinical Trials Transformation Initiative, Duke University, including Science Briefings from Perlstein Labs, Recursion Pharma and Tute Genomics.

Global Genes would like to congratulate the following 2015 RARE Champion of Hope award recipients:

RARE Champion of Hope – Patient Ambassador: Ben Huang

Ben was diagnosed with Spinal Muscular Atrophy (SMA) weak type I/II, a devastating progressive motor neuron disease with no cure, around age one. In November 2014, Ben was one of six to represent the US Junior Team in competition in the World Math Team Championship in Beijing. Ben won a gold medal, the only US Team gold medal, and placed 13 out of 270 junior competitors. During his 11 years of life Ben has gone through 4 major surgeries and needs assistance with sitting up, breathing, speaking, bathing, feeding and many other tasks.  Despite his incredible challenges, Ben has shown the world that patients can make an impact, contribute and make a difference.

RARE Champion of Hope – Teen Advocacy: Shira Strongin

Shira is battling Ehlers Danlos Syndrome, Postural Orthostatic Tachycardia and Complex Regional Pain Syndrome. She is an active advocate and positive role model for young adults. Since age 10, Shira has continuously advocated for the rare disease community by organizing her own charitable functions and fundraisers including “Miles to Smiles.” She created her own blog to give voice to the many teens facing rare disease issues, presented multiple conferences on “Advocating For Yourself,” and even lobbied the Senate for support funding for rare disease research. She has said that she wants to bring young kids together in a coordinated way and empower them to make a difference moving forward. She has coined the phrase, Patients Today—Tomorrow’s Leaders, and hopes that it will represent the culture of her teen advocacy efforts and encourage those to get involved.

RARE Champions of Hope – Awareness: Andrew Lenchewski – Executive Producer, Michael Rauch – Producer, Writer, Director & Mark Feuerstein, Actor (Royal Pains)

Andrew, Michael, Mark and their team working on the USA Network’s original hit series, Royal Pains, have made a positive impact and difference in the rare disease community with their ability to bring awareness through the powerful reach of broadcast and major media outlets. As the show’s producer and lead actor, Andrew, Michael and Mark, have worked to bring awareness by writing into the series rare disease specific elements.  Royal Pains airs Tuesdays at 10 pm PST, 8 pm CT, 10 pm EST on USA Network.

RARE Champions of Hope – Brave Feats of Courage:

This year Global Genes is recognizing four amazing individuals that completed remarkable ‘Feats of Courage’ while working to raise awareness for rare disease.  

  • Noah Coughlan – 3x Transcontinental Racer and Rare Disease Activist
  • Courtnay Midkiff – Transcontinental Walker, Fabry Patient Activist
  • Jonathan Maidment – Appalachian Trial Hiker, Alpha-1 Patient Activist
  • Bonner Paddock – Kona Ironman Competitor, Marathoner, Extreme Climber, Author, Cerebral Palsy Patient Activist

RARE Champions of Hope – Advocacy: Marianna Palka, Producer and Patient Advocate & Lucy Walker, Director (The Lion’s Mouth Opens)

Marianna and Lucy will be honored for their work on the documentary “The Lion’s Mouth Opens.” The film is about the journey and the decision by a young woman (Marianna Palka) to find out through genetic sequencing if she has inherited the Huntington’s disease gene – The disease that has impacted her family over generations.  An incredible story and film that has won multiple awards and honors at many of the nation’s film festivals.

RARE Champions of Hope – Industry: Alexey Salamakha & Nicole Riley (Novartis Pharmaceuticals Corporation)

Alexey and Nicole work in Patient Advocacy at Novartis Pharmaceuticals Corporation and have dedicated much of their time and energy to helping patients and improving patient care.  Alexey and Nicole are not senior executives at this major pharma company, in fact, they are committed employees that are working tirelessly to bring innovative programs to rare disease patient communities to make a difference in their daily lives.  Their creativity, innovation and total commitment to the patients they serve is impressive and inspiring.

The fourth annual RARE Patient Advocacy Summit™ will take place the two days prior to the gala, September 24 – 25. Advocate honorees will be recognized at a special awards ceremony on Friday, September 25 and include:

RARE Champion of Hope – Science: David Fajgenbaum (Castleman Disease Collaborative Network)

Dr. David Fajgenbaum is the co-founder and executive director of the Castleman Disease Collaborative Network and is battling the condition himself. He was diagnosed with Idiopathic Multicentric Castleman Disease (iMCD) in 2010. In 2012 he accelerated research and treatment discovery for iMCD and is now a Research Assistant Professor of Medicine in the Division of Hematology/Oncology at the University of Pennsylvania. His research has initiated a paradigm shift in how doctors diagnose and treat iMCD. Dr. Fajgenbaum was recently recognized in Forbes Magazine’s 2015 30 Under 30 list for Healthcare.

RARE Champion of Hope – Advocacy: Alison Rockett Frase (Joshua Frase Foundation)

Alison is the founder and Vice President for the Joshua Frase Foundation. She has been working tirelessly for years to help find a treatment for Myotublar Myopathy and continues this work despite losing her son to this condition several years ago. Alison is also on the governing board of the Congenital Muscle Disease International Registry (CMDIR) that supports key infrastructure needed to work towards a treatment for rare muscle disorders. 

RARE Champion of Hope – Medical Care and Treatment: Dr. Maria Escolar (NDRD) presented by SERMO

Dr. Maria Escolar established the Program for Neurodevelopment in Rare Disorders at UPMC (NDRD) in 2002 to help children and families understand the impact of rare neurological disease in child development. Dr. Escolar is board-certified in neurodevelopmental disabilities, has 15 years of experience as a practicing clinician and researcher, has published several papers with respect to the results of stem cell transplantation patients and has performed research on MRI imaging to help assess Krabbe patients. Recently she raised funds to develop the Virtual Medical Home at Pittsburgh Children’s Hospital. This Virtual Medical Home has enabled her to care for patients across the world, reaching children from 27 different countries and in 47/50 US states. 

SERMO is the United States’ top-ranked social network for fully verified, licensed physicians with nearly 400,000 doctors and is now available for doctors in seven countries: the US, UK, Canada, Australia, South Africa, Ireland and New Zealand. Founded in 2005, SERMO’s mission is to unite physicians and provide them with a safe, private and trusted platform for free and open discussions. 

RARE Champion of Hope – International Teen Advocacy: Madi Vanstone

Madi is a young rare disease advocate from Canada with Cystic Fibrosis. At just 12 years old, Madi successfully advocated for the funding of Kalydeco, the drug that treats Cystic Fibrosis, in Canada. She was awarded Youth Advocate award from the Canadian Cystic Fibrosis foundation and was recognized for helping have the drug funded in other provinces across Canada. She also was recognized by CORD for her efforts with a Youth Advocacy Award.


RARE Champions of Hope – Collaborations in Advocacy: Bill Strong & Victoria Strong (theGSF)

Bill and Victoria Strong started the Gwendolyn Strong Foundation (theGSF) and the non-profit apparel brand, NEVER GIVE UP, in 2009 after their first daughter, Gwendolyn, was diagnosed with Spinal Muscular Atrophy (SMA). Using technology, social media and smart partnerships theGSF focuses on funding and advancing research, advocating on Capitol Hill and providing technology and critical assistance grants to those facing SMA. Since 2009, they have funded 22 research grants, spearheaded rare disease legislation signed by President Obama, granted 225 iPads and spread awareness to tens of millions around the world, while working collaboratively with multiple SMA organizations and other advocacy stakeholders.

RARE Champions of Hope – Collaborations in Science: Ben Lenail, Kathleen O’Sullivan and Dr. Florian Eichler –  ALD Connect

ALD Connect is a patient-powered research consortium focused on the neuro-metabolic disease X-linked adrenoleukodystrophy (X-ALD). Less than two years ago, Dr. Florian Eichler challenged the ALD patient advocacy and research community with the goal to rapidly improve the quality of life for ALD patients/families and eventually eradicating the disease entirely. From this thought, the clinical trial network, ALD Connect, was created.  An Executive Committee made up of eleven members, who are one-quarter patients and patient advocates, govern ALD Connect.  Kathleen is an advocate from Boston, MA who will be recognized for bringing a depth of experience and a unique perspective to the governance. Ben Lenail is a founding officer and is currently Director of Business Development at Alta Devices, a solar energy company in Sunnyvale. Ben is also a member of HealthTech Capital; an investment group focused on healthcare IT.

RARE Champion of Hope – Advocacy: Lisa Genova (Inside the O’Briens & Still Alice)

Lisa Genova graduated valedictorian, summa cum laude from Bates College with a degree in Biopsychology and has a Ph.D. in Neuroscience from Harvard University. She is the author of the New York Times bestselling novels Still Alice, Left Neglected, Love Anthony, and Inside the O’Briens.  Lisa’s storytelling contributes to the understanding and important roles of rare diseases and disorders, and in particular with Huntington’s disease in her newest novel, Inside the O’Briens. In addition to her writings, Lisa has spoken about the neurological diseases with her appearances on the Today Show, Dr. Oz, the Diane Rehm Show, CNN, Chronicle, Fox News, Canada AM and was featured in the Emmy award-winning documentary film, TO NOT FADE AWAY. 

“Global Genes congratulates all of the inspiring nominees and recipients of the 2015 RARE Champions of Hope awards,” Nicole Boice, CEO Global Genes commented. “We are so fortunate to have the opportunity to honor, acknowledge and thank so many rare disease champions from around the globe who are proving that great accomplishments can be achieved by dreaming, planning, taking action and believing.”

The gala and summit annually raise awareness for over 7,000 different types of rare diseases that affect roughly 30 million Americans and approximately 350 million people worldwide. Proceeds from the evening will benefit Global Genes’ educational programs, as well as the organization’s new Patient Impact Grant program. The program offers support groups and charities that care for patients affected by rare disorders the opportunity to secure funding for projects that make a tangible difference in their disease communities.

To date, sponsors of the 2015 Tribute to Champions of Hope Gala and RARE Patient Advocacy Summit include the following organizations:

Premiere Sponsors:

Alexion, Amicus, Healthcare at Home, Walgreens, Genzyme and Shire.

Other Sponsors include:

Aegerion Pharmaceuticals, Abeona Therapeutics, Alnylam Pharmaceuticals,​

Ambry Genetics, Audentes, BIO, BioMarin, Biogen, Counsyl, Dohmen Life Science Services, Every

​L​ife Foundation for Rare Diseases, ​GSK, Horizon Pharma, Illumina, Insmed, Intercept, Idis, Invitae, ​

Jazz Pharmaceuticals, New Enterprise Associates, ​Novartis Oncology, Pfizer, Recordati Rare Diseases, Retrophin, Raptor Pharmaceuticals, SERMO, Shire, Sigma-Tau Pharmaceuticals, Ultragenyx and Vertex.

About Global Genes – Allies in Rare Disease

Global Genes is a leading global rare disease patient advocacy organization. The group’s mission is to eliminate the challenges of rare disease, by providing patients with educational tools, building awareness, providing critical connections and support to people and resources, and through investment in technologies that will positively impact affected patients and families. Recognized worldwide by the Blue Denim Genes Ribbon™, Global Genes unites experts, advocates and patients of all ages to stand together in hope for treatments and cures for the estimated 7,000 rare and genetic diseases that impact approximately 30 million Americans and over 350 million people worldwide.




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