The latest news from Israeli scientists, published in the Times of Israel, is that researchers at Tel Aviv University have accurately targeted cancerous cells in mice using CRISPR.…
On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…
In a recent press release, gene therapy company Orchard Therapeutics ("Orchard") announced that the FDA approved Orchard's Investigational New Drug (IND) application for OTL-200. This gene therapy solution is…
According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…
From November 13-16, researchers, doctors, and others participated in the American Association for the Study of Liver Diseases (AASLD)'s Annual Meeting, or The Liver Meeting Digital Experience. During the…
In late November, RNAi therapeutics company Alnylam Pharmaceuticals ("Alnylam") announced that its therapy OXLUMO (Lumasiran) received approval from the European Commission (EC) for both pediatric and adult patients with…
When gene therapy company Asklepios BioPharmaceutical, Inc. ("AskBio") and biotechnology company Selecta Biosciences ("Selecta") joined forces to combat genetic disorders, each company brought its best technology and insights. AskBio…
Takeda Pharmaceuticals has recently awarded XOMA Corporation $2 million for reaching the next milestone in the development of mezagitamab, a treatment for myasthenia gravis (MG). According to Biospace, XOMA has…
According to a press release from the biopharmaceutical company Sanofi, the company's investigational treatment avalglucosidase alfa will have its Biologics License Application (BLA) evaluated by the US Food and Drug…
According to an article from Biospace, Takeda Pharmaceuticals has released the results from their Phase 3 HELP trial, an extension study of TAKHZYRO. This hereditary angioedema (HAE) treatment has shown…
According to a story from Fierce Pharma, the drug company Alnylam Pharmaceuticals has seen its investigational therapy lumasiran (marketed as Oxlumo) recently gain approval for use in the EU as…
According to a story from PR Newswire, the pharmaceutical and diagnostics company The Menarini Group recently announced that its drug tagraxofsup (marketed as Elzonris) has a received a positive opinion…
The annual meeting of the American College of Rheumatology was recently held, where data was presented on Cimzia and Bimekizumab. Not only were both of these therapies found to benefit…
According to Biospace, Acceleron Pharma has presented preliminary data from their ongoing Phase 2 trial of sotatercept, a treatment for pulmonary arterial hypertension (PAH). The study, titled SPECTRA, is designed…
According to a story from GlobeNewswire, the rare disease liver company Albireo Pharma, Inc., recently announced positive results from a phase 3 study testing the company's experimental drug odevixibat as…
This year, the American College of Rheumatology (ACR) held their Convergence 2020 meeting virtually from November 5-9, with additional poster presentations on November 10 and 21. According to a…
According to a press release from the biopharmaceutical company Sanofi, the Biologics License Application (BLA) for its drug avalglucosidase alfa has been accepted by the US Food and Drug Administration…
Syndax Pharmaceuticals has plans to release data on axatilimab, a treatment for chronic graft-versus-host disease (cGVHD), at the 62nd American Society of Hematology (ASH) Annual Meeting, which will take place…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy, or SMA, is a genetic disease that affects an individual’s central and peripheral nervous systems, as well as their…
Early last week, Agios Pharmaceuticals ("Agios") announced that its treatment Mitapivat received Orphan Drug designation. This orally administered therapy is a pyruvate kinase R (PKR) activator. As Agios has…
Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…
Rhythm Pharmaceuticals, based in Boston, MA, is a biopharmaceutical company focusing on the development of therapies to treat rare genetic diseases that cause obesity. Until now, there have not been…
IntraBio has recently launched an extension study of their Phase II trial of IB1001, a treatment for Niemann-Pick disease Type C. This decision was made shortly after analyzing the positive…
Sierra Oncology has recently released two abstracts that will be presented at the 62nd American Society of Hematology (ASH) Annual Meeting. The meeting is planned for December 5-8. Both abstracts…
According to a story from Newswise, the genome editing company Intellia Therapeutics has recently announced that the first patient has been dosed in its phase 1 trial, which will test…
© 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.
