In early January 2021, biopharmaceutical company Avidity Biosciences ("Avidity") announced updates from its 2021 pipeline. Currently, Avidity is working to create a line of Antibody Oligonucleotide Conjugates (AOCs), therapeutic options…
Parent Project Muscular Dystrophy's (PPMD) goal is to end Duchenne muscular dystrophy (DMD), and they've just made another step in the right direction. They have recently announced the launch of…
The medical community has been aware for some time now that patient involvement in his or her treatment is a critical part of therapeutic development. Biopharmaceutical companies acknowledge their obligation…
According to a story from Globe Newswire, the genetic medicine company Sarepta Therapeutics has announced top-line data from part one of its clinical trial. This trial is investigating its experimental…
A webinar hosted by CureDuchenne featuring an informative program by Mesentech is scheduled for 4:30pm EST on Thursday, December 10th. The community is invited to join in and learn directly…
Mesentech and CureDuchenne have announced a partnership that includes generous funding from the Charles H. Hood Foundation. The partnership has developed a targeted therapy for reversing bone wastage in boys…
Solid Biosciences has just reported that the FDA has finally lifted the hold they had placed on their Phase I/II trial for Duchenne muscular dystrophy (DMD). This trial is called…
Parent Project Muscular Dystrophy (PPMD) is a nonprofit that focuses on fighting for a cure, better resources, and more awareness for Duchenne muscular dystrophy (DMD). More than a year ago…
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…
According to a story from Newswise, trial results released in the journal PLOS Medicine suggest that the drug vamorolone could provide an enhancement in the effectiveness of corticosteroid therapy in patients…
This year, at the 2020 CNS-ICNA Conjoint Meeting, researchers identified ataluren as a potential treatment that inhibits disease progression for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Research suggests…
According to CureDuchenne, two companies have began a collaboration in an effort to create new gene therapies for Duchenne muscular dystrophy (DMD). Ultragenyx and Solid Bioscience are both using their…
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…
While many pharmaceutical companies submit approval and marketing applications, not all are successful. According to Fierce Biotech, a subsidiary of Questex, Santhera Pharmaceuticals ("Santhera"), is one of the most recent…
According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…
According to Medical Xpress, there may be a new treatment option for patients with Duchenne muscular dystrophy (DMD). Traditionally, DMD is treated with glucocorticoids. However, these often require high doses…
As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…
Sarepta Therapeutics has met with the Office of Tissues and Advanced Therapies of the FDA in order to discuss a Type C written response about a clinical trial for SRP-9001,…
Happy Friday! Sept. 13-19 is Balance Awareness Week. It's intended to bring attention to balance-related vestibular disorders such as Meniere’s disease and vestibular migraine. Looking for ways to help out?…
Happy Friday! Monday, Sept. 7 is Duchenne Muscular Dystrophy Awareness Day. DMD is characterized by the inability to make the protein dystrophin in muscles and can result in heart and…
Earlier this week, Sarepta Therapeutics announced that the FDA approved a New Drug Application (NDA) for Casimersen (SRP-4045). The treatment, for patients with exon 45 amenable Duchenne muscular dystrophy (DMD),…
by Lauren Taylor from In The Cloud Copy Duchenne muscular dystrophy (DMD) is a rare genetic condition that ultimately leads to the wasting away of muscles over time. The condition…
According to BioSpace, the first patient has been enrolled in a trial of pamrevlumab, a Duchenne muscular dystrophy treatment. The third phase of LELANTOS plans to evaluate the drug's effects…
As society becomes increasingly technological, researchers are getting closer and closer to mapping the entire human genome. In facts, shares Medical XPress, we may be nearly there. While many…
According to a story from the CureDuchenne Blog, Dr. Kevin Flanigan of Nationwide Children's Hospital has recently dosed two Duchenne muscular dystrophy patients with an experimental gene therapy. This gene…
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