In a late May 2023 news release from clinical-stage biotechnology company Acepodia, the company shared that the first patient has been dosed in a Phase 1 study. Within this…
Ventilatory assistance, NG tubes, tracheostomy: all of these may be used to care for infants born with bronchopulmonary dysplasia (BPD), a chronic lung disease that most commonly affects preterm…
The AACR Annual Meeting 2023 took place from April 14-19. During the meeting, stakeholders from across the cancer community—from patients to clinicians—come together to discuss trends, research, and clinical practice.…
On May 10, 2023, reports EMPR, the U.S. FDA approved Elfabrio (pegunigalsidase alfa-iwxj) for adults who are living with Fabry disease. Developed by Protalix Biotherapeutics in conjunction with Chiesi Global…
Within the Phase 3 APHENITY study, researchers worked to determine the efficacy of sepiapterin in both adults and children with phenylketonuria (PKU). Formerly known as PTC923, sepiapterin was developed…
In a late April 2023 news release, biotechnology company UNITY Biotechnology, Inc. ("UNITY") shared that the company had observed positive long-term follow-up data from the Phase 2 BEHOLD trial. Within…
According to an article published in Cold Agglutinin News, Dianthus Therapeutics (“Dianthus”) is planning on holding a proof-of-concept study in 2024. Within the study, the company would evaluate the safety…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Currently, there are no cures for amyotrophic lateral sclerosis (ALS). Symptomatic therapy, alongside disease-modifying treatments like Rilutek, Radicava, and Relyvrio, may be used to manage this condition. However, there…
According to a story from Endpoints News, the drug developer Incyte has opted to put an end to its phase III clinical trial evaluating parsaclisib, an orally available PI3K inhibitor,…
In a news release from mid-April 2023, clinical-stage biotechnology company Abivax SA shared that 2-year results were available from a Phase 2b open-label maintenance study evaluating obefazimod for individuals…
In a news release on the company’s website, biopharmaceutical company Applied Therapeutics, Inc. shared that positive data was available from the Phase 3 ACTION-Galactosemia Kids trial. Within the trial, researchers…
Our blood has different clotting factors, or protein, that helps clot and stop bleeding after injuries. But when you don't have enough of these proteins, the blood can't clot…
Revolo Biotherapeutics was founded with one specific goal in mind: to benefit people living with autoimmune and allergic diseases by creating novel, life-changing therapeutics that induce long-term remission. They…
People living with bronchiectasis, a chronic condition characterized by damaged airways, experience progressive lung damage and inflammation. This can lead to a multitude of consequences, including a reduction in…
STATHMIN-2 (STMN2) is a protein that has been proven to play a role in axonal development, repair, and stability. This protein is highly expressed in human motor neurons; research…
Data from the first cohort of the Phase 2 KEYNOTE-158 trial, evaluating pembrolizumab for individuals with advanced thyroid carcinoma, showed positive safety and efficacy. According to Cancer Network, 103 participants…
The Muscular Dystrophy Association (MDA) recently held its MDA Clinical & Scientific Conference in March 2023. During the conference, stakeholders in the community discussed research trends and clinical practices associated…
The American Academy of Dermatology (AAD) held its Annual Meeting from March 17-21, 2023. During the meeting, stakeholders from the dermatology community connected to discuss clinical practices, research trends, and…
Contributed by Critical Path Institute (C-Path) and republished by Patient Worthy - find the original source article here. C-Path’s CDRC is positioned to inform future clinical trials for diseases of…
Right now, there are a number of therapeutic options for people living with Waldenstrom macroglobulinemia (WM), such as chemotherapy, immunotherapy, radiation, plasmapheresis, stem cell transplantation, and targeted therapy. However, these…
Currently, an estimated 47-50% of global nasopharyngeal carcinoma cases occur in China, with the regions of Guangdong, Guangxi and Fujian being heavily affected. There are a number of treatment…
In a news release from commercial-stage biopharmaceutical company Calliditas Therapeutics AB CALT ("Calliditas"), the company shared positive topline results from the Phase 3 NefIgArd study. Within the study, researchers…
When Cheryl Navar decided to have a child, in-vitro fertilization (IVF) seemed like the right move. Navar knew that she had a cancer gene and didn’t want to transfer…
In August 2021, the U.S. Food and Drug Administration (FDA) approved Opdivo (nivolumab) for the adjuvant treatment of a rare form of cancer called urothelial carcinoma. More particularly, the drug…
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