BridgeBio Pharma Receives FDA Fast Track Designation for Investigational Therapy for the Treatment of Limb-girdle Muscular Dystrophy Type 2I.
15 Sep, 2021
A fast-track designation facilitates the development and review of drugs meant to treat a serious condition with an unmet medical need. FDA will review each request and make a decision within 60 days based on whether the drug fills an unmet medical need in a serious condition.
An Open Letter to Patients and Their Families from Douglas M. Sproule MD MSc Chief Medical Officer, ML Bio Solutions - George Mclendon, CEO ML Bio Solutions - Neil Kumar CEO BridgeBio Pharma
The rapid spread of COVID-19 globally has led to tremendous disruption in the health care industry. We are actively assessing the impact the virus will have on our work at ML Bio Solutions, including the timing of our natural history study and subsequent clinical trials. We are doing our absolute best to minimize any delays, but we know that right now health care institutions and doctors around the world have shifted their focus to emergent COVID-19 cases. Many hospitals and physicians have postponed elective medical procedures and delayed the start of new studies. There is disruption across the country and globe as people are ordered to stay home to slow the spread of the virus. We support these steps, which are needed to minimize the devastating impact of this global pandemic.
Please know that despite these new hurdles we all face, our commitment to patients and their families does not change. That commitment is the cornerstone of our mission at ML Bio Solutions. We exist to bring meaningful medicines to patients as quickly and as safely as possible. But we do have to work within the new reality that is emerging. We are looking at the rapidly changing healthcare landscape on an hour-by-hour basis and trying to minimize disruption to our programs as much as possible.
We are in this together. There will inevitably be delays and we don’t know what the future holds, but we will not stop working as fast as we possibly and safely can for patients. You have our word.
Stay safe.
Douglas Sproule, MD MSc, CMO ML Bio Solutions - George McLendon, CEO ML Bio Solutions - Neil Kumar, CEO BridgeBio Pharma
What about the price of treatment!
3 Jun, 2019
The Institute for Clinical and Economic Review reflected on the prices of certain treatments for Duchenne Muscular Dystrophy. Because the treatment economics in LGMD2I are subjected to even more stress, I think this article may be of interest to you. 2019 ICER Concerns on Cost-effectiveness of DMD Gene Therapies
Complementary Genetic Testing!
23 Jan, 2019
Sanofi Genzyme has partnered with PerkinElmer Genomics to offer a complimentary genetic testing program called The Lantern Project. This program will provide diagnostic testing for several disorders including Limb-Girdle Muscular Dystrophy and Other Proximal Muscle Weakness.
Announcement
Deflazacort study in people with LGMD2I
23 Jan, 2019
A clinical study of Deflazacort (Emflaza®) in people with LGMD2I has started. This is an advanced study that hopes to enroll 100 adults to test the effects of the steroid on mucle function. Participants will receive deflazacort 0.6 milligrams per kilograms per day (mg/kg/day) orally for 52 weeks.
The study is sponsored by PTC Therapeutics and has clinical trial sites in the USA, Canada, Denmark, France, Germany, Norway, the Russian Federation, and Sweden. more info about this study can be found at Study information.
We made another post in our section Clinical Studies in LGMD2I
FDA announcement about Losartan
23 Jan, 2019
Losartan Potassium Tablets, USP and Losartan Potassium and Hydrochlorothiazide Tablets, USP by Torrent Pharmaceuticals: Recall Due to the Detection of N-nitrosodiethylamine (NDEA) was added to the FDA Recalls webpage.
Announcement
Nebula Genomics: A Start up to sequence your whole genome
15 Nov, 2018
Nebula Genomics wants to build a genomic and health data marketplace for consumers, researchers and the medical community. Nebula may end up providing your entire genome sequence for free, and let you own the data. Though, it seems that there is a fee in order to start the application process. A free, full genome sequence may be available to you if researchers and pharma companies find your genome sequence interesting. Nebula is the latest comer to the field of health data marketplace. Please do thorough research if you are interested in having your genome sequenced. Announcement
A new animal model for LGMD2I may help identify corrective drug compounds
20 Sep, 2018
The authors have developed a zebrafish model of LGMD2I in order to screen large library of chemical compounds for potential corrective drugs. Zebrafish have proven to be a powerful tool for studying muscle function owing to their high numbers of offspring, low maintenance costs, evolutionarily conserved muscle functions, and the ability to rapidly take up small molecular compounds during early larval stages. Article
Pfizer Terminates Domagrozumab (PF-06252616) Clinical Studies for the Treatment of Duchenne Muscular Dystrophy
30 Aug, 2018
Pfizer had sponsored a phase 1/2 clinical trial to test the safety and the benefit of treating patients with LGMD2I. The LGMD2I program was an extension of an ongoing program in Duchenne Muscular Dystrophy (DMD). On August 30, Pfizer announced the termination of the Phase 2 safety and efficacy study and the open-label extension study because results of the Phase 2 safety and efficacy study did not show efficacy as measured by the 4-Stair Climb test after one year of treatment. Press Release
Ribitol Could Benefit Patients With Rare LGMD and Related Diseases
27 Aug, 2018
A recent study published in the journal Nature Communications highlights a potential therapy for the treatment of LGMD2I. In this study, a group of researchers tested dietary complementation of LGMD2I mice with pure ribitol. The results suggested that adding high quantity of ribitol to the drinking water of the mice significantly lowered fibrosis in the skeletal muscles and in the heart and diaphragm muscles. Press Release and Article
MDA Funds Creation of LGMD Clinical Research Network to Speed Therapy Development
7 Jul, 2018
In its latest round of grants, MDA committed funding to develop clinical research tools and infrastructure for LGMD. The grant, totaling $700,000 over two years, will support a network of seven MDA Care Centers with expertise in LGMD research and clinical care. The objective is to facilitate the development of tools and infrastructure needed to efficiently and effectively conduct clinical trials and accelerate treatments for LGMDs. Press Release
Treatment of Tamoxifen and Raloxifene provides benefit to mice affected by lgmd2I
4 Apr, 2018
The authors observed that after long–term treatment with either Tamoxifen or Raloxifene mice showed increased strength and endurance and a decrease in muscle fibrosis. Article