Catalyst Pharmaceuticals expects to report 2019 net product revenues of $102 million for Firdapse (amifampridine), the first and only therapy available for treating the symptoms of Lambert-Eaton myasthenic syndrome (LEMS). In addition, the biopharmaceutical company anticipates 2020 Firdapse net product revenues of $135 million to $155…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
The HealthWell Foundation is offering financial assistance to eligible Lambert-Eaton myasthenic  syndrome (LEMS) patients in the United States to help with treatment costs. The new fund, which assists with insurance copayments and premiums, provides up to $12,000 in a yearlong grant to those who…
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…
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