National Organization for Rare Disorders (NORD) Announces 2024 Rare Impact Award Honorees, Emmy Award-Winning Journalist, Peter Alexander to Host

QUINCY, Mass., April 22, 2024 /PRNewswire/ -- The National Organization for Rare Disorders (NORD(®)) will honor a distinguished group of individuals and industry leaders for their contributions to help improve the lives of more than 30 million Americans with a rare disease at the annual Rare Impact Awards. NBC News Chief White House Correspondent Peter Alexander will reprise his role as host and emcee at this year's event which will take place on June 8 in Universal City, California at 6:00 p.m. PDT.

"This year, we are thrilled to celebrate with the West Coast rare disease community for the first time, and to acknowledge the monumental strides made by our honorees and industry innovators, including our first Youth Champion Award," says Peter L. Saltonstall, President and CEO of NORD. "Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."

The awards ceremony, to be held at Universal Studios Hollywood immediately following NORD's signature patient and family conference, the Living Rare, Living Stronger NORD Patient and Family Forum, will spotlight the exceptional contributions of rare disease advocates, researchers, healthcare professionals, and companies. The honorees are an extraordinary group that includes pioneers in genetic research, patient advocacy leaders, and innovative companies whose FDA-approved treatments have significantly improved patient outcomes. Each honoree's story is a testament to the power of innovation and perseverance.

Emcee Peter Alexander regularly reports on the deeply personal story of his sister Rebecca, who has Usher Syndrome, type III -- the leading genetic cause of progressive deaf-blindness: "I am honored to emcee the Rare Impact Awards again this year," says Alexander. "Having witnessed my sister's journey, I know the challenges rare disease families face are not just physical but also emotional, mental, and financial. This community's strength is inspiring, and I look forward to meeting this year's honorees and celebrating their hard-earned triumphs."

Tickets for the Rare Impact Awards are available at rareimpact.org.

Rare Impact Award Recipients

    --  Yann Le Cam: Lifetime Achievement Award recipient, Yann Le Cam, is
        celebrated for his three-decade-long history of advocacy in rare
        diseases, significantly impacting European regulations and global rare
        disease policy through his leadership at EURORDIS-Rare Diseases Europe.


    --  The Hemophilia Foundation of Southern California (HFSC): Winner of the
        Abbey S. Meyers Leadership Award, the HFSC has been pivotal in providing
        comprehensive support and fostering substantial community engagement
        among the Spanish-speaking population affected by bleeding disorders.


    --  The Honorable Gus M. Bilirakis of Florida: Recognized with the Policy
        Changemaker Award for his unwavering support in the U.S. House of
        Representatives, Representative Bilirakis (FL-12) is honored for
        championing multiple pieces of critical legislation that enhance access
        to treatments and support the rare disease community.


    --  The Honorable Liz Reyer of Minnesota: Also a Policy Changemaker Award
        recipient, Representative Reyer (MN-52A) is honored for her significant
        contributions to the rare disease community in Minnesota, including her
        efforts in establishing and serving on the Minnesota Rare Disease
        Advisory Council.


    --  Dr. Jose Abdenur of California: Recognized as a Medical & Scientific
        Trailblazer, Dr. Abdenur's extensive work in metabolic disorders at
        California's CHOC Children's Hospital and his leadership roles in
        various genetic screening programs mark his profound impact on rare
        disease diagnosis and treatment.


    --  Jana Monaco of Virginia: A Community Champion honoree, Jana Monaco's
        experience with her children--a son diagnosed with isovaleric acidemia
        as a toddler and a daughter diagnosed with the same condition in
        utero--led her to years of advocacy that influenced newborn screening
        policies, significantly impacting early diagnosis and treatment of rare
        diseases in Virginia.


    --  Irfan Patel of Delaware: Recognized as a Community Champion, Irfan
        Patel's dedication to enhancing awareness and support for rare disease
        communities in Delaware through legislative advocacy and community
        engagement was inspired by two of his children who are living with a
        rare metabolic disorder.


    --  Darlene Shelton of Missouri: Another Community Champion honoree, Darlene
        Shelton of Missouri, has spearheaded initiatives to reform state and
        national emergency medical protocols for individuals with special
        medical needs through her nonprofit, Danny's Dose, named in honor of her
        grandson who has hemophilia.
    --  Abigail Villarreal of Texas: Nominated as NORD's first ever Youth
        Champion, Abigail Villarreal has created an annual event, "Be Rare
        Spirit Day," in recognition of Rare Disease Day to increase awareness
        and education about rare diseases among her peers and within her
        community in Texas.

Industry Innovators

In addition to the individual award recipients, the Rare Impact Awards are also honoring companies that have developed treatments changing the lives of those with rare diseases. These innovative companies include:

    --  Biogen for Skyclarys(®): The first treatment to improve neurological
        function and slow disease progression in those living with Friedreich's
        ataxia, a rare progressive neurogenetic disease.


    --  BioMarin for Roctavian(TM): The first gene therapy for adults with
        severe hemophilia A, reducing the need for ongoing routine therapy.


    --  Chiesi Global Rare Diseases for Lamzede(®): The first enzyme
        replacement therapy approved in the U.S. for the treatment of
        non-central nervous system manifestations of alpha-mannosidosis in
        adults and pediatric patients.


    --  Ipsen Biopharmaceuticals for Sohonos(TM): An oral medication that is the
        first treatment to reduce the volume of extra-skeletal bone formation in
        adults and children with fibrodysplasia ossificans progressiva (FOP).


    --  Pharming for Joenja(®): The first approved treatment for people
        affected by Activated PI3K Delta Syndrome (APDS), a rare primary
        immunodeficiency.


    --  Regeneron Pharmaceuticals for Veopoz(TM): The first and only treatment
        indicated specifically for CHAPLE disease, a rare hereditary immune
        disease.


    --  SpringWorks Therapeutics for Ogsiveo(TM): The first approved treatment
        for adults with progressing desmoid tumors who require systemic
        treatment.


    --  Travere Therapeutics for Filspari(TM): The first and only
        non-immunosuppressive therapy to reduce proteinuria in adults with
        primary IgA nephropathy (IgAN) at risk of rapid disease progression.
    --  Vertex Pharmaceuticals for Casgevy(TM): A groundbreaking genome editing
        therapy for patients 12 years of age or older with sickle cell
        disease.Top of Form

Attendees of the Rare Impact Awards are also invited to attend the Living Rare, Living Stronger NORD Patient and Family Forum earlier that day, which offers educational programming designed to help patients, families, and caregivers navigate the complexities of living with a rare disease. Registration information can be found at livingrare.org.

About the National Organization for Rare Disorders (NORD)

With a 40-year history of advancing care, treatments and policy, the National Organization for Rare Disorders (NORD) is the leading and longest-standing patient advocacy group for the more than 30 million Americans living with a rare disease. NORD, a 501(c)(3) nonprofit, is dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 340 patient organization members, is committed to improving the health and well-being of people with rare diseases by driving advances in care, research and policy. For more information, please visit rarediseases.org.

About Emmy Award-winning Journalist, Peter Alexander
Peter Alexander is co-anchor of SATURDAY TODAY and Chief White House Correspondent for NBC News. He reports for all platforms of NBC News and MSNBC, including NBC Nightly News with Lester Holt, TODAY, Meet the Press and NBC News Now.

Rare Disease Facts & Impact:
https://rarediseases.org/wp-content/uploads/2024/04/Rare-Disease-Fact-Sheet.pdf

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SOURCE National Organization for Rare Disorders (NORD)