Patricia Simon Patricia Simon was born with a cleft lip and palate. She is now a nurse, a public speaker, a patient advocate, and a writer. She has a great…
According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European…
According to a story from PR Newswire, a new collaboration between the Hairy Cell Leukemia Foundation (HCLF) and the Leukemia & Lymphoma Society (LLS) will aim to support critical research…
According to a story from Charcot-Marie-Tooth News, CMT UK, a United Kingdom-based nonprofit focused on Charcot-Marie-Tooth disease, has released a series of seven exercise videos tailored to patients as part…
New Trial A Phase 1A trial for GMA301 was completed this year in Australia for pulmonary arterial hypertension (PAH). This trial demonstrated a positive safety profile for a range of…
A recent study, published in the journal Medicine, has found that Mycopheenolate mofetil (MMF) could be a more advantageous therapy for lupus nephritis than cyclophosphamide (CYC) when it comes to improving serum complement…
According to a story from PR Newswire, the biopharmaceutical company reVision Therapeutics, Inc. has just announced that its experimental therapy REV-0100 has received both Rare Pediatric Disease designation and Orphan…
According to a story from Biotech 365, the biotechology company Kezar Life Sciences, Inc. recently announced that its experimental therapy KZR-616 has earned Orphan Drug designations from the US Food…
According to a story from SMA News Today, the Patient Access Network (PAN) Foundation announced that it is now offering financial assistance to help patients living with the rare disease…
Researchers at the Mayo Clinic have collaborated with the international community to develop a potential way to test for spinocerebellar ataxia type 3 (SCA3), also called Machado-Joseph Disease. Additionally, they…
A recent study published in PLOS ONE has reinforced the importance of vaccinations specifically for those already fighting a rare disease. This study was written by Sylvie Lesage from the…
A new treatment for Duchenne muscular dystrophy (DMD) has recently been approved by the FDA. The therapy is called viltolarsen and it was created by Nippon-Shinyaku and the National Center…
A recent study has found a 12.5% improvement for patients with severe COVID-19 when baricitinib was used in combination with remdesivir. That means a faster recovery time for patients and…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently announced that it has awarded grant funding that will go towards six clinical trials that will…
In mid-October, biopharmaceutical company Zogenix announced that its Dravet syndrome treatment FINTEPLA (fenfluramine) received a positive CHMP opinion. The CHMP is part of the European Medicines Agency (EMA). While…
According to a story from BioSpace, the biopharmaceutical company Kite announced recently that the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, has released…
According to Biotech365, the National Hemophilia Foundation (NHF) recently revised its treatment recommendations for patients with congenital fibrinogen deficiency, or Factor I deficiency. Now, intravenously administered fibryga, developed by…
According to a story from Vice World News, the Solanki family, who are residents of Ahmedabad, the largest city in India's western state of Gujarat, recently took action to save…
In a recent press release the US Food and Drug Administration (FDA) has officially approved remdesivir (marketed as Veklury) as a treatment for cases of COVID-19 that require hospitalization. It…
According to a story from BioSpace, the biotechnology company Ascentage Pharma announced that two of its investigational treatments have recently received Orphan Drug designation from the US Food and Drug…
According to a story from BioSpace, the gene therapy company Axovant Gene Therapies Ltd. recently announced that it has been given Rare Pediatric Disease designation from the US Food and…
by Lauren Taylor from In The Cloud Copy Cervical dystonia, sometimes called spasmodic torticollis, is a condition in which the affected individual’s neck muscles involuntarily contract which causes the head…
According to a story from GlobeNewswire, the medical device company SoniVie announced recently that the US Food and Drug Administration (FDA) has granted approval for its Investigational Device Exemption (IDE).…
One of the biggest issues that many individuals fighting severe COVID-19 face is an overactive immune response. In effort to protect itself, the body creates dangerous levels of inflammation. Then,…
About one month ago, the HealthWell Foundation announced the beginning of the Adrenal Insufficiency Fund. Designed to assist patients with adrenal insufficiency, the fund will grant financial assistance to…
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