XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. All clinical trials rely on a well-oiled machine of motivated professionals, but nowhere is this more true than in advanced-therapy studies in rare diseases where knowledge is sparse.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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pharmaphorum

OCTOBER 16, 2020

By definition, however, trial master files represent a much richer and more detailed source of data on a drug and how it performs. For instance, provided a drug has not failed a trial on safety, the side effects it caused in one population could constitute on-target effects in another.

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XTalks

OCTOBER 16, 2024

Hympavzi’s approval highlights the broader need for innovative hemophilia treatments.

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XTalks

FEBRUARY 28, 2022

Related: UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD. Below are some facts and information about rare diseases, including rare disease clinical trials and orphan drugs. How is a Rare Disease Defined? Are Most Rare Diseases Genetic?

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XTalks

SEPTEMBER 27, 2024

In a news release published yesterday, Pfizer announced it will voluntarily withdraw its sickle cell disease (SCD) therapy Oxbryta (voxelotor) from all markets worldwide. The company said it is also discontinuing all active clinical trials for voxelotor and expanded access programs around the world.

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XTalks

SEPTEMBER 22, 2022

Clinical Trial Behind Spevigo. The efficacy of Spevigo (spesolimab) was determined based on the results of the Effisayil 1 double-blind, randomized, Phase II clinical trial. Spevigo is the first approved treatment option for GPP in adults.

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XTalks

NOVEMBER 27, 2023

Throughout the course of extensive clinical trials, Pombiliti and Opfolda have consistently exhibited a favorable safety profile, and patients have generally tolerated the treatment well.

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XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). He added that, “A deep body of evidence now points to the potential for durable effects in HSC gene therapy for certain severe genetic diseases including MLD.

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XTalks

AUGUST 29, 2022

It is difficult to control where the viral vectors insert genes in the genome, and it is difficult to manufacture large quantities of clinical-grade viral vectors. Scientists have previously shown that they could replace small sections of the IL2RA gene where mutations occur in patients.

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Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

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XTalks

SEPTEMBER 16, 2024

Related: FDA’s New Rare Disease Innovation Hub to Elevate Patient Care The NDA submission was supported by data from the KONFIDENT Phase III clinical trial and ongoing KONFIDENT-S open-label extension trial.

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XTalks

NOVEMBER 10, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio The FDA approval of Adzynma was based on its demonstrated safety and efficacy in a Phase III global cross-over study, the first of its kind for cTTP. Adzynma’s approval is the second for Takeda this week.

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XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. Nulibry Trial Results. The efficacy of Nulibry in the treatment of MoCD Type A was demonstrated in data from three different clinical trials that was compared to data from a natural history study.

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The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. The impact of CNS diseases extends beyond patients—to their families and society as well.”

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XTalks

MAY 12, 2023

What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. So, although it’s not a genetic treatment for Rett syndrome, it does act on the core pathophysiology behind Rett syndrome, which is that synaptic loss.

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XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

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XTalks

APRIL 15, 2021

million people in the US, yet there is no treatment for the most common form of the disease: heart failure with preserved ejection fraction (HFpEF). A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl. Heart failure affects about 6.2

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XTalks

SEPTEMBER 17, 2020

Acromegaly Surrogate Endpoint: Serum growth hormone and serum insulin-like growth factor 1 (IGF-1) are acceptable surrogate endpoints for acromegaly clinical trials involving somatostatin analogs such as octreotide, lanreotide and pasireotide. The traditional approval pathway can be pursued by meeting this surrogate endpoint.

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XTalks

MARCH 21, 2024

MLD is a rare genetic disease affecting the brain and nervous system caused by a deficiency of the enzyme arylsulfatase A (ARSA), which results in the accumulation of sulfatides (fatty substances) in cells. The transaction has proven fruitful with the milestone US approval of the gene therapy just a few months later.

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XTalks

OCTOBER 1, 2020

The drug is currently in clinical trials for alcoholic hepatitis (AH), nonalcoholic steatohepatitis (NASH), as well as in COVID-19 patients with acute liver or kidney injury. Working on finding therapies for genetic diseases at NeuBase, Dr. Sooter says, “Everyday, we get up and that’s our goal and it really drives everyone.

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XTalks

OCTOBER 8, 2024

Scholar Rock announced positive results from the Phase III SAPPHIRE trial evaluating apitegromab, an investigational muscle-targeted therapy, in patients with spinal muscular atrophy (SMA). SMA is a genetic neuromuscular disease causing progressive muscle weakness and loss of motor function.

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The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Of course, this is how biotech investments work.

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