Editor's Note: Patient Worthy is honored to share this article from our friend Jessica Lynn. To see the article in its original format, please click here. When five-year-old Bennett hit…
A decade of accumulated clinical experience with nusinersen has culminated in a significant regulatory advancement. According to Pharma Times Online, Biogen's newly approved high-dose Spinraza regimen represents a thoughtful evolution…
Patient Worthy is excited to share our most recent episode of our podcast Wait, How Do You Spell That? featuring Lisa Batista. Lisa shares her story with us of living with…
Kevan Chandler, was born with a rare, and progressive neuromuscular disease: spinal muscular atrophy (SMA). At age 33, he needs assistance with almost all activities of daily living, from eating…
A remarkable story shared by Sportskeeda illustrates how a moment of frustration on the football field turned into hope for a family in need. In March 2021, after a controversial…
Spinal muscular atrophy (SMA) is a rare disease causing progressive muscular weakness. Severe forms of the disease can be fatal to infants. Apitegromab is an experimental therapy that was successful…
The month of August is recognized as Spinal Muscular Atrophy (SMA) Awareness Month. This is a time of year in which the community works to spread awareness about this rare…
Testing for rare conditions can be costly and time-consuming. In India, testing for spinal muscular atrophy (SMA), a rare genetic disorder, has traditionally cost between 3500 INR to 7000 INR…
For decades researchers have had to isolate segments of genes in order to study them. They called the isolated gene a “mini gene.” Ravindra Singh, a biomedical science professor at…
₹17.5 crores—or $2,102,128. That's the cost of medication for a young boy named Kanav, who lives in Najafgarh, a town in the Southwest Delhi district. Kanav is living with…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
When Adrienne Vollmer first learned that her son Graham had spinal muscular atrophy (SMA), she was shocked. At the time, there were no FDA-approved treatments (three now exist). The life…
Each summer advocates for rare diseases take advantage of longer daylight hours and use the opportunity to showcase the hopes, needs, and wants of people with rare diseases such as,…
Orphan drug designation was first introduced to the European Union in 2000. The designation is granted to therapies intended to treat, diagnose, or prevent a rare, life-threatening, or chronically…
John knew from an early age that he not only loved music but had confidence in his musical ability. However, there was another very troubling issue in his life. John…
In March 2023, 14-year-old Rylie Erbacher visited Washington, D.C. for a one-of-a-kind experience. The teen, who has spinal muscular atrophy (SMA) type 2, was one of just 10 people nationally…
You caught me: I'm a Swiftie. I first saw Taylor Swift on her Speak Now tour and, in 2 weeks, am taking on the Eras tour in Philadelphia (PS: if you have any…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
For the first year of his life, Celebi Remillard and his mother Tatyana frequently took the drive from Klamath Falls to Portland to visit his care team. When Celebi was…
After Tabitha Wright heard the news from the doctor, her heart dropped. Her daughter Aspen had just been diagnosed with spinal muscular atrophy (SMA) type 1, a rare genetic…
August has begun and you know what that means – it is Spinal Muscular Atrophy (SMA) Awareness Month! During this month, many patients, advocacy groups, family members, and others work…
In clinical trials evaluating Zolgensma for children with spinal muscular atrophy (SMA), the maximum weight of participants was 18.7 pounds. However, parents with children weighing over that weight might be…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
What type of cognitive changes are present in individuals with spinal muscular atrophy (SMA) type 3? More so, do these cognitive changes affect clinical factors and, if so, how? According…
Altogether, there are multiple subtypes of spinal muscular atrophy (SMA), a rare genetic disorder causing muscle weakness and generation. Four months ago, Aniya Porter was born with type I, a…
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