PTC Therapeutics Inc. has just announced that their supplemental new drug application for Evrysdi (risdiplam) has been given priority review by the FDA. The aim is to expand the indication…
Rare disease therapies are often difficult to access, whether that is due to price, a patient's location, or another factor. Because of these obstacles, many rare disease patients find themselves…
When Rhonda Stevey met her grandson, Koehyn, she was immediately smitten. The grandmother, who lives in Ohio, felt like her grandson was the happiest infant she had ever met. Unfortunately,…
According to the Daily Sabah, the Health Ministry of Turkey is taking a new approach to family planning and screening for genetic disorders. As of October 8, 2021, the Health…
According to YAHOO News, twenty-two-month-old Devdan has a rare disease called type 2 spinal muscular atrophy (SMA2) which affects nerve cells that control muscles. If left untreated progressive muscle weakness…
The month of August is recognized as Spinal Muscular Atrophy (SMA) Awareness Month. This is a time of year in which the community works hard to spread awareness about this…
Access to treatment is an issue for patients across the world, especially for those with rare diseases. Laurie Young, who was diagnosed with type 2 spinal muscular atrophy (SMA) at…
Spinal muscular atrophy is rare progressive muscular disease that over time, erodes a patient’s movement. Without early intervention, the condition erodes the young patients ability to breathe and even swallow.…
According to a story from bbc.com, five month old Arthur was born with the rare disease spinal muscular atrophy (SMA). He is one of the first people in the UK…
The U.S. Food and Drug Administration (FDA) runs a number of programs designed to facilitate research and drug development for serious conditions. One such program is the Fast Track program;…
As reported by CNN; Tabitha Haly still manages to make time for her life-long craft as a singer-songwriter and live performer while acting as a vice president at JP Morgan…
Becoming a new parent is always scary. There are plenty of worries and concerns: setting up a nursery, making the right decisions, and of course, the baby's health. Samantha and…
Spinal muscular atrophy (SMA) is a rare, genetic disorder that impacts the muscles, making them progressively weaker over time. To combat this muscle atrophy, patients must exercise their muscles to…
Spinal muscular atrophy (SMA) patients aged two months and older will now have an at-home treatment option in Europe. The European Commission (EC) has approved of Evrysdi for those who…
Sometimes it can be difficult to find or afford certain medications designed for your condition. However, this may soon be changing for Canadians with spinal muscular atrophy (SMA). On March…
There's positive news for Evrysdi, a treatment for spinal muscular atrophy (SMA). The EMA Committee for Medicinal Products for Human Use (CHMP) has recommended that the drug be approved, and…
Jason Kraft, the owner of Electric Bike Technologies, has brightened the lives of two sisters with two electronic tricycles that are typically made for senior citizens. Maggie and Charlie Monnin,…
As reported in WTNH; in Stafford Springs, CN, the Pokorny family was rudely awakened to the world of rare diseases when their new daughter Olivia was born with spinal muscular…
Turkish citizens are putting pressure on their government in regards to access to rare disease treatments, specifically spinal muscular atrophy (SMA) therapies. There are currently two drugs available to Turkish…
Spinal Muscular Atrophy Spinal muscular atrophy (SMA) is a rare disease that causes progressive muscle loss and resultant muscle weakness. It is caused by a lack of motor neurons. It…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a disease that occurs due to loss of motor neurons, which are the nerve cells that…
Alina Citovic was diagnosed with spinal muscular atrophy at age five, just three years ago. At the time of her diagnosis, Spinraza was already approved by the FDA. While the…
According to a story from SMA News Today, a recent case study has reported instances in two spinal muscular atrophy (SMA) patients in which repeated administrations of the therapy nusinersen…
Ever since he was a young boy, JR Trout, who lives with spinal muscular atrophy (SMA), had a sense that there was something different about him. For years, he never…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a disease that affects the patient’s central nervous system, peripheral nervous system, and skeletal muscles or…
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