Georgia Bio Advocacy Blog

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BioMarin Pharmaceutical commemorates 5 years of helping children with fatal rare disease

April 29, 2022

CLN2 Batten disease is a rare, inherited, fatal disorder that primarily affects the nervous system as a result of a failure to produce enough of the enzyme TPP1. The signs and symptoms of this condition typically start between the ages of 2 and 4, featuring recurrent seizures and coordination difficulty. BioMarin Pharmaceutical in San Rafael…

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Saol Therapeutics and InformedDNA Partner to Offer Genetic Counseling to Patients with Rare Mitochondrial Disease

April 28, 2022

Program Discusses Access to a Pivotal Phase 3 Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency (DCA/PDCD trial; NCT02616484) Saol Therapeutics, a company researching new treatments for rare diseases, is pleased to announce a partnership with InformedDNA®, the nation’s leading applied genomics solutions company, to offer genetic counseling for the families of individuals with a…

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CMS Decision is an Enormous Setback for Alzheimer’s Patients and a Dangerous Infringement on FDA’s Scientific Autonomy

April 8, 2022

Patients will suffer because of this bureaucratic and unnecessary second-guessing of the FDA, says BIO CEO. Dr. Michelle McMurry-Heath, President and CEO of the Biotechnology Innovation Organization (BIO), released the following statement in response to the Centers for Medicare and Medicaid Services’ (CMS’s) National Coverage Determination (NCD) to restrict coverage for drugs used to treat…

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Potential drug for Alzheimer’s disease could block early damage, MCG researcher says

April 1, 2022

Most drugs proposed for Alzheimer’s disease target the gunk that accumulates in the brains of patients but fail to take enough away to make a difference or improve outcomes, said the head of the new Alzheimer’s Therapeutics Discovery program at Medical College of Georgia. It would be better to target a mechanism much earlier in…

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Stephanie and Christopher’s Story: Rare Disease and Medical Nutrition

February 28, 2022

Any parent would agree that having a child changes your world forever. Yet, having a child with a rare disease throws you not only into the role of parent but also advocate, educator, researcher, and medical provider. Those with rare diseases, and the guardians’ that care for them, have different perspectives on the world. We…

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Nathan’s Story: VCP Disease and the need for a Rare Disease Advisory Council in Georgia

February 28, 2022

Since I was a freshman at Georgia Tech, I have had a clock ticking in my head.  Since my mother presented symptoms of the VCP gene mutation, I had grown up knowing that a rare disease existed in my family and that there was a 50% chance that I might get it as an adult…

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American Red Cross: Celebrate Black History Month by Participating in a ‘Sleeves Up’ Campaign

February 7, 2022

February is Black History Month – a time to honor the significant achievements of Black Americans while making an impact today. This year’s theme focuses on the importance of Black health and wellness. The American Red Cross is honoring the legacy of Dr. Charles Drew, an African American surgeon, and researcher who pioneered new methods for…

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Alzheimer’s patient groups protest U.S. Medicare coverage proposal limiting use of new drugs

January 24, 2022

Alzheimer’s patient groups, disappointed by Medicare’s plan to sharply limit coverage of new drugs for the brain-wasting disease, are planning publicity and lobbying campaigns to protest a proposal they say could delay their use for 10 years. “Congress has to know how bad this will be for patients,” said John Dwyer, president of Global Alzheimer’s…

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Patient’s Corner: Living with Chronic Illness Vol. 3

October 4, 2021

By Dorothy Leone-Glasser Breast cancer survivors face many challenges while going through treatment and in post treatment.  They experience a wide range of physical, mental, emotional and spiritual pain. There are lingering side effects to treatment that are long term and can be hard to manage. Many times, the fear of what will happen is overwhelming.  Cancer…

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Patient’s Corner: Living with Chronic Illness Vol. 2

September 13, 2021

By Dorothy Leone-Glasser I met Mapillar Dahn and the MTS Sickle Cell Foundation, Inc. at our Rx in Reach GA Coalition Capitol Day. She was excited to participate in our event and had a table set to distribute Sickle Cell Awareness material and to engage visitors, attendees and legislators on the challenges of living with…

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