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,…
The FDA has recently accepted NeoImmuneTech's Investigational New Drug (IND) application for NT-17, a treatment for progressive multifocal leukoencephalopathy (PML). Now that the application has been cleared, the biopharmaceutical company…
The medical community has been aware for some time now that patient involvement in his or her treatment is a critical part of therapeutic development. Biopharmaceutical companies acknowledge their obligation…
In a late December 2020 press release, biopharmaceutical company Cerecor Inc. (“Cerecor”) announced the FDA acceptance of an Investigational New Drug Application (IND) for CERC-007. The human monoclonal antibody (mAb)…
The Orphan Drug Act originated in 1983 as an incentive for pharmaceutical companies who worked on developing treatments for rare disease. Since rare diseases are well, rare, choosing to work…
COVID-19’s tentacles have invaded the New Drug Applications (NDAs) for 2020. A recent article in Fierce Pharma reported that the FDA has been especially vigilant in scrutinizing NDAs this year,…
The FDA has officially approved remdesivir for the treatment of adults and children (over the age of 12 and weighing at least 40 kg) who have been hospitalized for COVID-19.…
This week, the US FDA announced that they approved the drug Nucala (mepolizumab) to treat patients with hypereosinophilic syndrome, a rare chronic blood disease which causes organ damage. Nucala is…
In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…
According to biotechnology Gilead Sciences, the FDA rejected a new drug application (NDA) for filgotinib. The drug, designed to treat patients with moderate to severe rheumatoid arthritis (RA), is known…
This past week, biopharmaceutical company Kiniksa Pharmaceuticals announced that their drug, rilonacept, received Orphan Drug Designation from the FDA. Following positive data from the RHAPSODY Phase 3 clinical trial,…
Biotechnology company Talaris Therapeutics, Inc. submitted an Investigational New Drug (IND) application to the FDA for their novel cell therapy treatment FCR001. As per a press release, the FDA…
In a July 9 press release, the Familial Chylomicronemia Syndrome (FCS) Foundation announced its support for a bill introduced by Congressman Paul Tonko (D-NY) and David B. McKinley (R-WV).…
As shared in Medscape, the FDA recently approved oral octreotide (Mycapssa) for the treatment of patients with acromegaly. These delayed-release capsules are the first approved oral somatostatin analog ever approved.…
Stacey Saiontz is many things: a worker, a wife, a mother. But right now, she is concerned. As an allergy parent, or a parent whose child has severe food…
According to a drug filing and press release from Pharmaceutical Business Review, the FDA granted a priority review to GlaxoSmithKline for their humanized monoclonal antibody therapy, Nucala (mepolizumab). Currently,…
A gene therapy for the treatment of GM1 gangliosidosis was granted Orphan Drug Designation in April. PBGM01 seeks to treat the most severe form of this condition - infantile. Read…
A recent news release on BioSpace shares how GTX-102, an investigational antisense oligonucleotide drug therapy for Angelman syndrome, was granted Fast Track designation from the FDA. The therapy, created by GeneTx…
As originally reported in Yahoo Finance, the coronavirus is disrupting many of our expectations of how we planned to live out the next few months. Some frequenters of the hospitals…
Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…
According to a story from IFLScience, the US Food and Drug Administration (FDA) approved a total of 48 novel medicines last year. A further investigation of these drugs reveals that…
A big step forward for those with amyloidosis! The European Medicines Agency (EMA) granted Pfizer a positive recommendation for the approval of its treatment for amyloidosis, a rare disorder that…
By Caitlin Seida from In The Cloud Copy 2019 was a record year for drug recalls due to safety and quality concerns. It’s also been a record year for the…
According to a story from The People's Pharmacy, the US Food and Drug Administration (FDA) has given the green light for the use of generic drugs manufactured in India despite…
Contact: Britta Vander Linden bvanderlinden@everylifefoundation.org 917.604.6518 30 million Americans need more treatment and diagnostic opportunities https://everylifefoundation.org/rare-disease-community-calls-on-congress-fda-to-enact-life-saving-public-policy-solutions/ (Washington, D.C., December 4, 2019) Hundreds of rare disease advocates from around the country were brought to Washington,…
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