In a news release from May 18, 2021, biopharmaceutical company Catalyst Biosciences, Inc. ("Catalyst") shared that the first patient was dosed in the Phase 1/2 MAA-202 clinical trial. During…
Clinical trials are extremely beneficial in learning how new and burgeoning treatment options can benefit patients. At the same time, these trials also offer insight into where treatments may not…
According to an article in Clinical Trials Arena, Blueprint Medicine has been steadily and speedily recruiting participants for its Phase II PIONEER trial, which examines Ayvakit as a treatment for…
For many patients with diffuse large B-cell lymphoma (DLBCL), one first-line treatment is rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP). However, even aggressive chemotherapy is not always enough for patients…
Poly ADP ribose polymerase (PARP) inhibitors are targeted cancer therapies which block PARP, an enzyme. Normally, PARP plays a role in DNA repair. Thus, inhibiting PARP prevents cancer cells from…
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,…
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…
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…
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,…
According to MarketScreener, patients with endometrial cancer in Europe now have a new treatment option. Recently, the European Commission (EC) granted conditional marketing authorization to GlaxoSmithKline for their therapy JEMPERLI…
According to a recent news release, CTX001, a gene-edited therapy for patients with transfusion-dependent beta thalassemia, received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The treatment was…
Over the last 50 years, doctors have been working to improve clinical outcomes for patients with a rare but aggressive form of ocular cancer: uveal melanoma. However, there has been…
According to the Pharmacy Times, patients with chronic inflammatory demyelinating polyneuropathy (CIDP) now have a new and potentially more effective treatment option. After submitting its supplemental Biologics License Application (sBLA),…
The 2021 virtual American Academy of Neurology (AAN) Annual Meeting is taking place from April 17 through April 22, 2021. During the conference, experts and researchers are offering a…
According to News Medical, researchers from the University of Cincinnati presented findings on a potentially new and effective treatment option for Pompe disease at the virtual American Academy of Neurology…
In a recent press release, RNA-targeted therapeutics company Ionis Pharmaceuticals, Inc. ("Ionis") shared the initiation of a Phase 2/3 clinical trial evaluating ION373 for patients with Alexander disease (AxD). Currently,…
According to a recent news release, the FDA cleared an Investigational New Drug (IND) application for VIP152, a potential therapeutic option for patients with Richter syndrome and relapsed/refractory chronic…
Clinical trials can be beneficial in many ways. For one, these trials show researchers whether a treatment’s impact is durable and sustained. Determining this often requires some follow-up period. According…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
