In a news release from June 9, 2021, biotechnology company Vertex Pharmaceuticals, Inc. ("Vertex") shared that its therapy TRIKAFTA (elexacaftor/texacaftor/ivacaftor and ivacaftor) was approved for expanded use. Now, the treatment…
According to a story from the Bognor Regis Observer, 25 year old Kieren Rogers had his first stroke last August. It was a minor event, but he still needed to…
Early diagnosis and prompt treatment are absolutely necessary for the best outcomes in many rare disorders. Keratoconus, a rare disease that impacts the cornea, is no exception. It is best…
684 words, 9% matched vs 1,329 words, 4% matched Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong…
According to a story from mdlinx.com, a recent study has found that around 20 percent of sudden cardiac death cases in US adults can be linked to pathogenic arrhythmia syndromes…
As of June 7, 2021, an injectable treatment is now available for patients aged 1 month+ with paroxysmal nocturnal hemoglobinuria. The FDA recently approved Ultomiris (ravulizumab-cwvz) for these patients. Previously,…
A 2019 Survey Found 70% of Patients with NMOSD Relapsed Every Year. According to a recent article in China Daily, the patients interviewed for this article said that their…
In early June 2021, SPG302, an investigational therapy for patients with amyotrophic lateral sclerosis (ALS), received Orphan Drug designation from the FDA. According to ALS News Today, the treatment…
As reported in Biospace; the clinical-stage biotech company Nanoscope Therapeutics, Inc. has announced a resonating gene therapy success: all participants with advanced retinitis pigmentosa (RP) maintained vision improvements a year…
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