In late May 2023, clinical-stage biopharmaceutical company Sangamo Therapeutics, Inc. (“Sangamo”) shared that its product isaralgagene civaparvovc (ST-920) was granted Fast Track Designation by the FDA. This designation is…
“Three years ago, my lung function was 11% and I was hours from death, then I received the gift of life – which was Trikafta.” These are the words of…
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…
According to a story from CBS News Boston, Scarlett is just six years old and was recently hit with a devastating diagnosis: Batten disease. Unfortunately, very little can be done…
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…
A court in Zabbar, Malta found the parents of seven-year-old Victoria guilty of her death by negligence. The court also determined that the system had failed the child by…
A positive opinion is needed from the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), to begin marketing a drug. Once a positive…
Ticks can carry a number of disease-causing pathogens. If an infected tick bites a human, that human can then contract several illnesses. In many cases, people who contract these…
In Japan researchers at the Juntendo University have discovered that when ECHS1 enzyme variations of mitochondrial enoyl-CoA hydratase short chain 1 (ECHS 1) do not function properly, they cause…
WebMD recently carried a story about a young woman who, after nine years of wheelchair confinement caused by a rare metabolic disease with no name, was able to walk…
The FDA has approved the drug Ayvakit developed by the biotechnology company Blueprint Medicines for the treatment of indolent systemic mastocytosis. According to an article in BiopharmaDive, Ayvakit had been…
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…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
As clinicians and scientists continue to delve into RNA sequencing benefits, they are learning how RNA sequencing provides insights for people with rare genetic conditions. The learning experience will provide…
On May 18, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
Fierce Biotech recently carried an article about the death of Terry Horgan. Terry was a 27-year-old Duchenne muscular dystrophy (DMD) patient and the brother of the CEO of the…
The 91st European Atherosclerosis Society (EAS) Congress took place from May 21-24, 2023. During the Congress, many stakeholders discussed the latest developments in basic, translational, and clinical research into vascular…
The energy in the crowd at American Family Field was electric. Everybody sat on the edge of their seat, waiting for the game to begin. If you’ve never been to…
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…
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…
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