Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
In a recent news release, gene therapy company Taysha Gene Therapies ("Taysha") shared the publication of preclinical data on the company's drug candidate, TSHA-102. Currently, Taysha is exploring TSHA-102 as…
In China, the Conditional Approval process allows for new drugs to be marketed and developed based on an urgent and unmet need. For example, drugs which treat patients with rare…
Clinical trial data offers immense insight into a drug's safety, efficacy, and tolerability. But it can also share how combining two treatments can impact patient outcomes or whether a…
According to MedPage Today, data from a preliminary study evaluating SHR0302, an investigational Janus kinase 1 (JAK1) inhibitor, showed promise for treating patients with moderate-to-severe atopic dermatitis (AD). Following treatment,…
Patient-reported and patient-driven registries can be crucial in understanding rare diseases or cancers, driving research, and forming communities. Now, according to a recent press release, a new registry is forming,…
In a sense, Langerhans cell histiocytosis (LCH) has always been somewhat of a mystery for doctors. As a rare multi-system disorder, LCH can affect the skin, lungs, bone marrow, spleen,…
Hyperphagia, or an abnormally stronger hunger or desire to eat, is one of the characteristics of Prader-Willi syndrome (PWS), a rare genetic disorder. Because of this, patients with PWS often…
Recently, there have been a number of reports of mucormycosis, a rare but sometimes fatal fungal infection, spreading throughout hospitals in India. According to MSN, the fungal illness is becoming…
Just about one month ago, pharmaceutical company Secura Bio, Inc. ("Secura") shared that the company completed enrollment for the Phase 2 PRIMO clinical trial. Altogether, 101 patients enrolled. The…
Clinical trials can provide immense insight into potential treatment options and patient outcomes. Currently, biopharmaceutical company Protagonist Therapeutics, Inc. ("Protagonist") is working to gain insight into the efficacy of…
On March 6, 2021, biopharmaceutical company Altavant Sciences ("Altavant") shared that it had initiated its Phase 2b ELEVATE 2 clinical trial. During the trial, researchers will evaluate rodatristat ethyl ("rodatristat")…
According to BioSpace, Farxiga (dapagliflozin), a treatment for patients with chronic kidney disease (CKD) and a risk of progression, recently became FDA-approved. Developed by AstraZeneca, the therapy is specifically designed…
There are many reasons why medical research is important. It helps to amplify the patient voice, improve disease-related knowledge, and determine potential treatment goals. Additionally, medical research provides insight into…
Last April, 25-year-old Obang Odol Okello became infected with dracunculiasis, also known as Guinea worm disease. According to Thomson Reuters Foundation News, the father of two, who lives in Ethiopia,…
Recently, researchers sought to examine how Nucala (mepolizumab) affected disease progression in patients with eosinophilic granulomatosis with polyangiitis (EGPA), especially when used in conjunction with corticosteroids. According to ANCA Vasculitis…
According to MedPage Today, ursodeoxycholic acid significantly improves patient outcomes in relation to intrahepatic cholestasis of pregnancy. This rare liver disorder increases the risk of fetal issues, stillbirths, or preterm…
Since its inception, the Clinical Data Interchange Standards Consortium (CDISC) has worked to advance data standards to the highest possible quality. These standards are then used to make data more…
Have you been paying attention to the news? If not, you might have missed that INCRELEX (mecasermin) is now commercially available in Canada! In a news release, biopharmaceutical company…
When it comes to rare diseases, the diagnostic process can be long and arduous. On average, patients wait around 7 years for a diagnosis. Oftentimes, this also complicates treatment and…
In a recent news release, biotherapeutics company CSL Behring shared that the company received FDA approval to update the Hizentra label for patients with chronic inflammatory demyelinating polyneuropathy (CIDP). Within…
Recently, the European Commission (EC) approved a marketing authorization for ADV7103 (Sibnayal), the first approved treatment for patients with distal renal tubular acidosis (dRTA). In a news release, pharmaceutical company…
In a news release from April 2021, biopharmaceutical company Jazz Pharmaceuticals, Inc. ("Jazz") shared that the FDA accepted its supplemental New Drug Application (sNDA) for review in relation to Xywav,…
About six months ago, pharmaceutical company Takeda pledged $300M towards developing Arrowhead Pharmaceuticals' ("Arrowhead") ARO-AAT, an RNA-silencing treatment for patients with alpha-1 antitrypsin deficiency (A1AD). According to Fierce Biotech, the…
In a news release from May 1, 2021, Chiesi Global Rare Diseases, a business unit of the pharmaceutical company the Chiesi Group, shared its FDA acceptance of FERRIPROX (deferiprone). Altogether,…
For patients with chronic kidney disease (CKD), the diagnostic journey can be long and arduous. Unfortunately, many individuals are not diagnosed until later stages of the disease. As a result,…
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