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About 17,884 results
FDA approves Vyondys 53 for Duchenne muscular dystrophy subtype
https://www.mdedge.com/clinicalneurologynews/article/214420/neuromuscular-disorders/fda-approves-vyondys-53-duchenne?channel=37225
Jake Remaly, Clinical Neurology News

Dec 17th, 2019 - The Food and Drug Administration has granted accelerated approval to Vyondys 53 (golodirsen) to treat patients with Duchenne muscular dystrophy (DMD) who have a mutation of the dystrophin gene that is amenable to exon 53 skipping. About 8% of patients with DMD have this type of mutation.

Amylyx drug helps slow ALS progression in mid-stage study
https://www.reuters.com/article/us-amylyx-pharma-study/amylyx-drug-helps-slow-als-progression-in-mid-stage-study-idUSKBN1YL1KH

Dec 17th, 2019 - (Reuters) - Privately held drug developer Amylyx Pharmaceuticals Inc said on Tuesday its experimental therapy to treat amyotrophic lateral sclerosis (ALS) helped slow the progression of the fatal neurological disorder in a mid-stage study. ALS, also known as Lou Gehrig’s disease, is a rare disorder that attacks nerve cells responsible for controlling voluntary muscles, and over 6,000 Americans ...

Sarepta shares surge after surprise approval of Duchenne Muscular Dystrophy drug
https://www.reuters.com/article/us-sarepta-fda/sarepta-shares-surge-after-surprise-approval-of-duchenne-muscular-dystrophy-drug-idUSKBN1YG2SQ

Dec 13th, 2019 - (Reuters) - Shares of Sarepta Therapeutics Inc (SRPT.O) soared 31% on Friday after U.S. regulators shocked Wall Street by approving the company’s treatment for a muscle-wasting disorder less than four months after rejecting the drug over safety concerns. FILE PHOTO: The headquarters of the U.S. Food and Drug Administration (FDA) is seen in Silver Spring, Maryland November 4, 2009. REUTERS/Jason...

Pexidartinib Receives Category 1 Recommendation from NCCN
https://www.mdedge.com/sarcomajournal/article/214275/pexidartinib-receives-category-1-recommendation-nccn?channel=39313

Dec 13th, 2019 - Pexidartinib, the newly approved agent to treat patients with tenosynovial giant cell tumor (TGCT), received a category 1 recommendation from the National Comprehensive Cancer Network (NCCN) in the recent update of its Clinical Practice Guidelines in Oncology, Soft Tissue Sarcoma (Version 4. 2019).

Sarepta shares surge after surprise approval of DMD drug
https://www.reuters.com/article/us-sarepta-stock/sarepta-shares-surge-after-surprise-approval-of-dmd-drug-idUSKBN1YH1M1

Dec 13th, 2019 - (Reuters) - Shares of Sarepta Therapeutics Inc soared 32% on Friday after U.S. regulators shocked Wall Street by reversing their rejection of its muscle-wasting disorder therapy less than four months ago, to allow the drug’s immediate launch. The Food and Drug Administration in August declined to approve the Duchenne Muscular Dystrophy (DMD) treatment, Vyondys 53, citing safety concerns, includ...

Three Quick Tips for Managing Osteoarthritis
https://www.medscape.com/viewarticle/922004

Dec 12th, 2019 - This transcript has been edited for clarity. Matthew F. Watto, MD: As usual, we're coming in hot. This is the Curbsiders. I am Dr Matthew Watto. Here are my two wonderful cohosts, Dr Stuart Brigham and Dr Paul Williams. Tonight we are going to be talking about osteoarthritis (OA). We had a wonderful interview with Dr Tuhina Neogi, the chair of rheumatology at Boston University School of Medicin...

FDA OKs Golodirsen (Vyondys 53) for Duchenne Muscular Dystrophy
https://www.medscape.com/viewarticle/922586

Dec 12th, 2019 - The US Food and Drug Administration (FDA) has approved golodirsen injection (Vyondys 53, Sarepta Therapeutics), the first treatment for Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. Last summer, the agency declined to approve of the drug on an accelerated basis, sending a complete response letter (CRL) to the company citing concerns over t...

New ASH guideline: VTE prophylaxis after major surgery
https://www.mdedge.com/surgery/article/214157/thrombosis/new-ash-guideline-vte-prophylaxis-after-major-surgery?channel=39313
Andrew D. Bowser

Dec 12th, 2019 - ORLANDO – The latest American Society of Hematology guideline on venous thromboembolism (VTE) tackles 30 key questions regarding prophylaxis in hospitalized patients undergoing surgery, according to the chair of the guideline panel, who highlighted 9 of those questions during a special session at the society’s annual meeting. Andrew D.

FDA Okays First Newborn Screening Test for Duchenne MD
https://www.medscape.com/viewarticle/922551

Dec 11th, 2019 - The US Food and Drug Administration (FDA) has authorized the marketing of the first newborn screening test for Duchenne muscular dystrophy (DMD), the agency announced earlier today. The GSP Neonatal Creatine Kinase-MM kit (PerkinElmer) measures levels of creatine kinase (CK) from dried blood samples collected from a draw from an infant's heel 24 to 48 hours after birth. Although high levels may...

Reduced kidney function linked to fractures in older women
https://www.mdedge.com/clinicianreviews/article/214101/osteoporosis/reduced-kidney-function-linked-fractures-older-women
Bianca Nogrady

Dec 11th, 2019 - Moderate reductions in kidney function in older women are associated with an increased short-term risk of fractures, according to a study published in Osteoporosis International. However, the longitudinal, population-based cohort study did not find an association with fracture risk either in women older than 80 years or in those with worse kidney function.

Age, gender, and race influence treatment decisions for calcaneus fracture
https://www.mdedge.com/surgery/article/213959/orthopedics/age-gender-and-race-influence-treatment-decisions-calcaneus?channel=27970
Steve Cimino

Dec 10th, 2019 - A variety of socio-demographic variables – including residing in a lower-income zip code and lacking private insurance – can influence treatment decisions for orthopedic trauma patients, according to a new study on the management of calcaneus fractures. “While our study demonstrated socio-demographic disparities regarding utilization of open reduction and internal fixation of calcaneus fracture.

Novartis's $90 Mln Swiss Factory to Help Solve Cell Therapy Bottleneck
https://www.medscape.com/viewarticle/921927

Dec 9th, 2019 - ZURICH (Reuters) - Swiss drugmaker Novartis's new $90 million cell and gene therapy factory in northern Switzerland is on track to begin commercial production of treatments, including Kymriah for cancer and Zolgensma for spinal muscular atrophy, in 2020. The new gene and cell therapy factory, expected to employ 450 people, will allow the drugmaker to make its Kymriah treatment for European pati...

Updated gout guidelines: Don’t let kidney function dictate allopurinol dosing
https://www.mdedge.com/clinicianreviews/article/213863/gout/updated-gout-guidelines-dont-let-kidney-function-dictate?channel=53
M. Alexander Otto

Dec 9th, 2019 - ATLANTA – Soon-to-be-published gout guidelines from the American College of Rheumatology will recommend dosing allopurinol above 300 mg/day to get serum urate below 6 mg/dL, even in people with renal impairment. M.

What to do when the evidence is not conclusive
https://www.mdedge.com/familymedicine/article/213849/asthma/what-do-when-evidence-not-conclusive?channel=296
MDedge Family Medicine; John Hickner, MD, MSc

Dec 9th, 2019 - Family physicians try to base treatment decisions on the very best available evidence from randomized trials and other high-quality studies. Very often, however, the evidence is not conclusive.

Aspirin Plus a DOAC May Do More Harm Than Good in Some
https://www.medscape.com/viewarticle/922341

Dec 8th, 2019 - ORLANDO, Florida — Combining aspirin and direct oral anticoagulant (DOAC) therapy for the secondary prevention of venous thromboembolism (VTE) or the prevention of stroke associated with nonvalvular atrial fibrillation (NVAF) without a clear indication was associated with increased bleeding risks in a large registry-based cohort. The study, which involved a cohort of 2045 patients who were foll...

Aspirin plus a DOAC may do more harm than good in some
https://www.mdedge.com/emergencymedicine/article/213797/thrombosis/aspirin-plus-doac-may-do-more-harm-good-some?channel=39313
Sharon Worcester

Dec 8th, 2019 - ORLANDO – Combining aspirin and direct oral anticoagulant (DOAC) therapy for the secondary prevention of venous thromboembolism (VTE) or the prevention of stroke associated with nonvalvular atrial fibrillation (NVAF) without a clear indication was associated with increased bleeding risks in a large registry-based cohort. Sharon Worcester/MDedge News Dr.

Pruritic and Erythematous Rash Resembling Marks Caused by a Lashing
https://www.mdedge.com/dermatology/article/213774/contact-dermatitis/pruritic-and-erythematous-rash-resembling-marks-caused?channel=113
Ford M. Lannan, MD, Thomas R. Evans, DO et. al.

Dec 6th, 2019 - Shiitake Mushroom Dermatitis Given the scattered and erythematous 1- to 2-mm macules and patches in a flagellate pattern on the shoulders, back, and neck, the differential diagnosis included shiitake mushroom dermatitis, bleomycin-induced flagellate dermatitis, dermatomyositis flagellate erythema, excoriation disorder, dermatographism, and keratosis lichenoides chronica. On further questioning,.

The Week That Wasn't: Healing Honey, Restored Limb Function, Anti-Cancer Anthrax
https://www.medscape.com/viewarticle/922271

Dec 5th, 2019 - This week the internet was abuzz with tales of honey's healing power, a new solution for spinal cord injuries, and using anthrax against cancer. Here's why you didn't see those headlines on Medscape. Honey's Healing Power? It's feasible to use Manuka honey, spread in tiny amounts between layers of surgical mesh, to kill bacteria, a new study suggests. The mesh that facilitates soft tissue heali...

Systemic Lupus Erythematosus Highlights From the 2019 ACR Annual Meeting
https://www.medscape.com/viewarticle/920781

Dec 5th, 2019 - New data relevant to the treatment of systemic lupus erythematosus (SLE) were presented at the 2019 annual meeting of the American College of Rheumatology in Atlanta. Dr Michelle Petri, who heads the Johns Hopkins Lupus Center, discusses key findings, including the results of two important clinical trials on the anti–interferon receptor blocker anifrolumab. TULIP 1, the first of the two phase 3...

Novartis R&D boss says doesn't see big opportunity in oral SMA therapy
https://www.reuters.com/article/us-novartis-investors-sma/novartis-rd-boss-says-doesnt-see-big-opportunity-in-oral-sma-therapy-idUSKBN1Y91S0

Dec 5th, 2019 - FILE PHOTO: The company's logo is seen at the new cell and gene therapy factory of Swiss drugmaker Novartis in Stein, Switzerland, November 28, 2019. REUTERS/Arnd Wiegmann ZURICH (Reuters) - Novartis research head Jay Bradner said on Thursday the Swiss drugmaker is focusing efforts against spinal muscular atrophy (SMA) on gene therapy Zolgensma and retreating from oral therapy like its molecule...