Vital Science

FOXG1: A Mom’s Mission

Being the parent of a child with an ultra-rare disease can be a daunting challenge. Nasha Fitter is no different. Her daughter Amara is nonverbal and suffers from epilep

Cure EB: Healing the Wounds

Imagine giving birth to a baby girl and noticing pieces of skin missing from her body almost immediately.   For Sharmila Nikapota, this was the case with her firstborn,

Clear Scientific: Combatting Drug Overdose from Methamphetamine, Fentanyl, and Polydrug

Stimulant use in the United States has exploded into a nationwide crisis, as deaths and emergency room visits resulting from methamphetamine and cocaine use are rising at

Mission Therapeutics: Inhibiting DUBs to Halt Diseases

At Mission Therapeutics, Sarah Almond serves as their Head of Pharmacology, but she wears many hats to help develop potential treatments for neurodegenerative conditions.

Cure Rare Disease: Continuing a Legacy

When he founded Cure Rare Disease in 2018, Rich Horgan was primarily focused on finding a treatment for his younger brother Terry, who had an ultra-rare form of Duchenne

H-Guard: Improving Physical and Mental Health

For the 850 million people around the world suffering from chronic kidney disease, the grind of dialysis treatment can wear them down in more ways than one.      Dr. An

Checking Vitals: The Disruptors

Over the past 12 months, we have shared some remarkable stories from the scientific community, but three of the most impactful have come from “Disruptors” in their respec

TREND: The Online Community Making a Difference

When Maria Picone’s daughter was diagnosed with Prader-Willi Syndrome (PWS), she turned to the internet for answers.     What she found was an online community of careg

Strength in Numbers

Imagine a world where your child has a rare disease that’s so rare, it doesn’t have a name and only a handful of people around the world have a similar condition. It can

Emily Whitehead: Pioneer and Hero

18-year-old Emily Whitehead aspires to be a writer, but nothing could have prepared her for the plot twist that struck her as a child.     In the space of just 24 hours

Modernizing Drug Development

For decades, researchers have utilized animal models to help predict the effects of a drug compound in humans.     With recent advances in technology and the passage of

SCA3: A Family Affair

Spinocerebellar ataxia type 3 (SCA3) is a neuromuscular disease affecting just 50,000 people in the United States, including three generations of the Klassen family.    

Moving FAST to Treat Angelman Syndrome

The effects of Angelman Syndrome (AS) on young children are debilitating. From an early age, they face an uphill battle with this neurogenetic disease’s unrelenting sympt

Free for Life: The Story of n-Lorem Foundation

When patients face the diagnosis of a rare disease, they face a costly, uphill battle to develop a treatment for their condition… that is, if they ever do.     Dr. Stan

Evie's Story Revisited

Imagine being diagnosed with an incurable rare disease before ever being born. This is the reality that Evie faced when she was diagnosed with hypophosphatasia, a conditi

Neurogenesis: Regenerating Hope

Could it be possible to regenerate neurons in the human body?    It’s a question that has driven Dr. Wise Young for over 40 years. He’s dedicated his career to research

AviadoBio: Bold and Courageous

Recent advancements in research has led to the development of effective therapeutics to combat neurodegenerative diseases, giving patients a renewed sense of hope there w

Checking Vitals

As we close in on the end of 2022, we’ve had the privilege to interview some amazing people who are doing great work with drug discovery.    Join Gina Mullane and Todd P

A Father’s Race Against Time

Spastic Paraplegia 50 (SPG50) is a neurodegenerative and neurodevelopmental disorder that is known to affect only 80 people around the world, including just one person in

Project ALS: Fueled by Love

For Valerie Estess, finding a cure for ALS is personal. Her sister, Jenifer, was diagnosed with this crippling disease in 1998, which led to the founding of Project ALS,