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Top Muscular Dystrophy News News

Why a New Opioid Alternative Is Out of Reach for some Pain Patients
United States · United States

Why a New Opioid Alternative Is Out of Reach for some Pain Patients

67% Left coverage: 3 sources
Journavx is the first truly new painkiller approved by the Food and Drug Administration in more than 20 years. But the drug is expensive, and many people can't get it yet. (Image credit: Vertex Pharmaceuticals) ...See the Story
Taiho's late-stage Duchenne candidate fails trial
Education

Taiho's late-stage Duchenne candidate fails trial

100% Center coverage: 1 sources
Taiho has suffered a blow to its near-term pipeline after a drug candidate for Duchenne muscular dystrophy (DMD) missed the mark in a phase 3 trialSee the Story
Capricor plans for deramiocel resubmission after FDA rejection
Mississippi · Mississippi

Capricor plans for deramiocel resubmission after FDA rejection

Capricor Therapeutics will resubmit its application for U.S. Food and Drug Administration (FDA) approval of its cell therapy deramiocel to treat Duchenne muscular dystrophy (DMD)-related heart disease, following the agency’s decision to not accept the original filing. According to a company press release, the FDA issued a complete response letter (CRL) requesting additional clinical data, stating that the initial submission did not sufficiently …Read Article

Digging Deeper: Mechanisms of Progression and the Case for a Dual Therapeutic Approach

Panelists discuss how chronic neuroinflammation involves distinct mechanisms from acute relapses—including microglial activation, mitochondrial dysfunction, and iron deposition—necessitating dual therapeutic approaches that address relapsing and progressive disease components.Denmark · DenmarkSee the Story

Households caring for DMD patients face high costs: US survey

Households incur substantial costs to accommodate people with Duchenne muscular dystrophy (DMD), according to a survey of patient caregivers in the U.S. Costs, such as those for purchasing and/or modifying an accessible vehicle, relocating to or building an accessible home, modifying home entrances, and buying medical equipment, were particularly high for households caring for patients with more advanced disease and limited walking abilities. “T…United States · United StatesRead Article

What risk vs. reward looks like in the patient world

The reward is that the chocolate cake tastes delicious and leaves you with a nice feeling. The risk is that you just spiked your sugar intake and may gain a few calories that take time and effort to burn off. The risk is that you may get injured, but the reward is an unbelievable rush of adrenaline when you parachute out of a perfectly good airplane by choice — if that’s your thing, of course. All of us weigh risk and reward on a daily basis and…Mississippi · MississippiRead Article

Giving thanks that my 3 sons with DMD have one another

Summer vacation in the Midwest typically begins in mid-May, and kids return to school by mid-August. Here in Nebraska, that puts us halfway through summer break already. Like most moms, I’m feeling a mix of emotions: grief as I anticipate the end of these few precious weeks home with my children, and slight relief to have a break from sibling arguments and nonstop streams of dirty dishes from snacks snuck from the kitchen and extra wet towels th…Nebraska · NebraskaRead Article

Ifetroban continues to show cardiac protection in Phase 2 DMD trial

Ifetroban, Cumberland Pharmaceuticals’ investigational oral therapy, improved heart function and reduced cardiac damage biomarkers in people with Duchenne muscular dystrophy (DMD), according to new trial data. These findings from the Phase 2 FIGHT DMD trial (NCT03340675) were presented at the Parent Project Muscular Dystrophy (PPMD) annual conference, held recently in Las Vegas. Long-term safety and efficacy data are expected later this year. Cu…Mississippi · MississippiRead Article

Accepting Help Is Difficult for Me as a Mom and a Caregiver

Late afternoon sunlight drifts into the house, brightening the blue paint on the walls. The room feels cheery and welcoming. Wicker baskets hold soft blankets, pillows cushion the couch, and dogs are sleeping on the rugs. I love walking into this room, and every room, in my home, which I have carefully curated to provide a calm and cozy haven. I have seven children: Lexi, 24; Max, 19; Chance, 18; Rowen, 16; Charlie, 14; Mary, 10; and Callie, 3. …Spain · SpainSee the Story

An odd dream made me wonder how my disability affects others

I’m an early riser. As I mentioned in a past column, I like to wake up early, get dressed, make coffee, and start the day with some quiet prayer and devotion time. However, I’m also a very sound sleeper, with some help from melatonin, sleep medication, and a comfy mattress. From the time I fall asleep around 11 p.m. until about 5:30 a.m., I’m dead to the world. Some shoulder, hip, or knee pain may wake me up during those hours, but I usually man…Read Article

Considering New Duchenne Muscular Dystrophy Therapies: Excon Skipping and Gene Therapy

A panelist discusses how older DMD patients might benefit from newer genetic therapies including gene therapy and exon skipping, though eligibility depends on specific mutations and absence of pre-existing immunity to AAV vectors.Mississippi · MississippiSee the Story

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