Translating passion into execution: Cure Rare Disease's founder on the business of biotech
When a career path chooses you in the form of a pressing personal cause, passion and commitment must soon be followed by execution. And that means schooling up — quickly. I had heard the call of entrepreneurship early. I had a new Harvard Business School degree in hand at the same time my brother’s health was declining steeply due to Duchenne muscular dystrophy. His fatal degenerative neuromuscular condition wouldn't wait, and the lack of viable option s in the pipeline for his gene mutation required entrepreneurial thinking. The science would come. I was in Boston, after all, immersed in bioscience. And it has. (Sourc...
Source: bizjournals.com Health Care:Biotechnology headlines - February 26, 2021 Category: Biotechnology Authors: Rich Horgan Source Type: news
FDA OKs First'Targeted Treatment' for Rare DMD Mutation FDA OKs First'Targeted Treatment' for Rare DMD Mutation
The FDA has approved casimersen (Amondys 45) injection for treating patients with both Duchenne muscular dystrophy (DMD) and a rare mutation of the DMD gene.FDA Approvals (Source: Medscape Pediatrics Headlines)
Source: Medscape Pediatrics Headlines - February 26, 2021 Category: Pediatrics Tags: Neurology & Neurosurgery News Alert Source Type: news
FDA Approves Amondys 45 (casimersen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 45
CAMBRIDGE, Mass., Feb. 25, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Amondys 45... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - February 25, 2021 Category: Drugs & Pharmacology Source Type: news
For UCLA-based startup, new muscular dystrophy treatment is a personal mission
Courtney Young helped develop the gene therapy at the heart of a biotech startup,MyoGene Bio, when she was a doctoral student at UCLA from 2013 to 2018. But the kernel of the company ’s endeavor is older still — and definitively personal.In 2008, when Young was in high school, she found out that her cousin, then just a toddler, had been diagnosed with Duchenne muscular dystrophy. She made it her mission to cultivate the skills needed to provide better options for him and others who were suffering with the deadly muscle-wasting disease.“I directed my career toward working on Duchenne,” said Young, MyoGene Bio’s co...
Source: UCLA Newsroom: Health Sciences - February 23, 2021 Category: Universities & Medical Training Source Type: news
After the Nobel, what next for Crispr gene-editing therapies?
Hailed as the ‘molecular scissors’ that will allow us to rewrite our genes, the DNA tool is being trialled in treatments for everything from sickle-cell anaemia to cancerWhen last year ’s Nobel prize for chemistry was awarded tobiochemist Jennifer Doudna and microbiologist Emmanuelle Charpentier for their work in developing the technique of gene editing known as Crispr-Cas9 (pronounced “crisper”), headlines hailed their discovery as “molecular scissors” that would allow us to “rewrite the book of life” – with all the complicated ethical questions that ability raises. But much of the excitement has nothi...
Source: Guardian Unlimited Science - February 21, 2021 Category: Science Authors: Philip Ball Tags: Gene editing Medical research Genetics Biology Science Cancer Huntington's disease Sickle cell disease Source Type: news
Invitation to Roche ’s virtual event on 24-month data of Evrysdi in patients with type 2 or type 3 spinal muscular atrophy presented at the 2021 MDA virtual conference
We are pleased to invite investors and analysts to participate in our virtual event on Friday, 19 March, 2021, highlighting Roche data presented during the 2021 Muscular Dystrophy Association - MDA -Virtual Clinical and Scientific Conference, from 15th to 18th February. (Source: Roche Investor Update)
Source: Roche Investor Update - February 19, 2021 Category: Pharmaceuticals Source Type: news
The Hardship of Social Distancing When Touch Is a Lifeline
For me, and many others with physical disabilities, touch is not merely a luxury or a pleasure, but an aspect of my functionality, my basic survival. (Source: NYT Health)
Source: NYT Health - February 10, 2021 Category: Consumer Health News Authors: Sonali Gupta Tags: Touch (Sense) Muscular Dystrophy Quarantines Muscles Genetics and Heredity Coronavirus (2019-nCoV) Physical Therapy Disabilities Source Type: news
Early Promise for Therapy Against Duchenne Muscular Dystrophy
THURSDAY, Jan. 28, 2021 -- An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests.
The severe form of muscular dystrophy -- which affects about one in 3,500 males born each year in the United States --... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - January 28, 2021 Category: General Medicine Source Type: news
What Is the Best Treatment for Muscular Dystrophy?
Title: What Is the Best Treatment for Muscular Dystrophy?Category: Diseases and ConditionsCreated: 1/28/2021 12:00:00 AMLast Editorial Review: 1/28/2021 12:00:00 AM (Source: MedicineNet Kids Health General)
Source: MedicineNet Kids Health General - January 28, 2021 Category: Pediatrics Source Type: news
Enhanced oral uptake of exosomes opens cell therapy alternative
(Cedars-Sinai Medical Center) Cell-derived exosomes are effective in treating disease when mixed with the dominant protein in breast milk and given orally, a new Smidt Heart Institute study of laboratory mice shows. The findings, published in the peer-reviewed Journal of Extracellular Vesicles, could help develop new oral medications for treating patients with muscular dystrophy and heart failure. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 12, 2021 Category: International Medicine & Public Health Source Type: news
Science Saturday: Across time, place to find cures for inherited heart diseases
Five years ago, Jay Schneider, M.D., Ph.D., was running a lab at The University of Texas Southwestern Medical Center in Dallas. The lab focused on researching cures for the genetic muscular-wasting disorder, Duchenne muscular dystrophy. His wife, Alice Chang, M.D., had accepted an offer to work at Mayo Clinic in Rochester, Minnesota, two years before. The [...] (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - January 2, 2021 Category: Databases & Libraries Source Type: news
Digging deep for differences in Duchenne muscular dystrophy
(UT Southwestern Medical Center) A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchene muscular dystrophy (DMD) in humans. The findings, published online recently in PNAS, could lead to new treatments for this devastating degenerative disease and insights into factors that affect muscle development. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 21, 2020 Category: International Medicine & Public Health Source Type: news
FDA approves Roche ’s OCREVUS® (ocrelizumab) shorter 2-hour infusion for relapsing and primary progressive multiple sclerosis
Basel, 14 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved a shorter two-hour infusion time for OCREVUS ® (ocrelizumab), dosed twice-yearly for those living with relapsing or primary progressive multiple sclerosis (MS) who have not experienced any prior serious infusion reactions (IRs). The approval was based on data from the randomised, double-blind ENSEMBLE PLUS study.“More than 170,000 people with MS have been treated with OCREVUS - the only approved B-cell therapy with a twice-yearly dosing schedule - and it is the most prescribed MS med...
Source: Roche Media News - December 14, 2020 Category: Pharmaceuticals Source Type: news
FDA approves Roche ’s OCREVUS® (ocrelizumab) shorter 2-hour infusion for relapsing and primary progressive multiple sclerosis
Basel, 14 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved a shorter two-hour infusion time for OCREVUS ® (ocrelizumab), dosed twice-yearly for those living with relapsing or primary progressive multiple sclerosis (MS) who have not experienced any prior serious infusion reactions (IRs). The approval was based on data from the randomised, double-blind ENSEMBLE PLUS study.“More than 170,000 people with MS have been treated with OCREVUS - the only approved B-cell therapy with a twice-yearly dosing schedule - and it is the most prescribed MS med...
Source: Roche Investor Update - December 14, 2020 Category: Pharmaceuticals Source Type: news
Muscle cell secrets
(Max Delbr ü ck Center for Molecular Medicine in the Helmholtz Association) A muscle fiber consists of just one cell, but many nuclei. A team at the MDC led by Professor Carmen Birchmeier has now shown just how varied these nuclei are. The study, which has been published in Nature Communications, can help us better understand muscle diseases such as Duchenne muscular dystrophy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 11, 2020 Category: International Medicine & Public Health Source Type: news
